HEALEY ALS Platform Trial News and Webinars

July 20, 2023: Regimen G, DNL343 Mechanism of Action & Science

Merit Cudkowicz, MD, MSc along with regimen co-lead Suma Babu, MBBS, MPH, and research scientists from Denali Therapeutics, presented the rationale and science behind DNL343, the investigational product being tested in Regimen G of the HEALEY ALS Platform Trial, and answered questions from the audience.
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March 27, 2023: Regimen F, ABBV-CLS-7262 Mechanism of Action & Science

Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD, along with regimen co-lead Senda Ajroud-Driss, MD and research scientists from Calico Life Sciences, presented the rationale and science behind ABBV-CLS-7262, the investigational product being tested in Regimen F of the HEALEY ALS Platform Trial, and answered questions from the audience.
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March 10, 2022: Regimen E, Trehalose Mechanism of Action & Science
Sabrina Paganoni, MD, PhD scientists from Seelos Therapeutics, and regimen co-leads Merit Cudkowicz, MD, MSc and Shafeeq Ladha, MD presented the rationale and science behind trehalose, the investigational product being studied in Regimen E of the HEALEY ALS Platform Trial, and answered questions from the audience.
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January 21, 2021: Regimen D, Pridopidine Mechanism of Action & Science

Merit Cudkowicz, MD, MSc, Sabrina Paganoni, MD, PhD, research scientists from Prilenia Therapeutics and regimen co-leads Jeremy Shefner, MD, PhD and Björn Oskarsson, MD, FAAN presented the rationale and science behind pridopidine, the investigational product being tested in regimen D of the HEALEY ALS Platform Trial and answered questions from the audience.
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February 4, 2021: Regimen C, CNM-Au8 Mechanism of Action & Science

Merit Cudkowicz, MD, MSc, Sabrina Paganoni, MD, PhD, research scientists from Clene Nanomedicine and regimen co-leads James Berry, MD, MPH and Nicholas Maragakis, MD presented the rationale and science behind CNM-Au8, the investigational product being tested in regimen C of the HEALEY ALS Platform Trial and answered questions from the audience.
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February 18, 2021: Regimen B, Verdiperstat Mechanism of Action & Science

Merit Cudkowicz, MD, MSc, research scientists from Biohaven Pharmaceuticals and regimen co-leads, Jinsy Andrews, MD, MSc and Suma Babu, MD, MPH, MBBS presented the rationale and science behind verdiperstat, the investigational product being tested in regimen B of the HEALEY ALS Platform Trial, and answered questions from the audience.
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February 25, 2021: Regimen A, Zilucoplan Mechanism of Action & Science

Merit Cudkowicz, MD, MSc, research scientists from UCB and regimen co-leads Sabrina Paganoni, MD, PhD and Christina Fournier, MD presented the rationale and science behind zilucoplan, the investigational product tested in regimen A of the HEALEY ALS Platform Trial, and answered questions from the audience.
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January 4, 2024: Weekly Q&A 

Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD of Massachusetts General Hospital provided an overview of the HEALEY ALS Platform Trial to date, presented news and updates, and answered questions from the audience.
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December 14, 2023: Weekly Q&A and EAP Update with James Berry, MD, MPH
James Berry, MD, MPH and Merit Cudkowicz, MD, MSc of Massachusetts General Hospital presented news and updates about Expanded Access in the field of ALS research. Dr. Berry provided an overview of ongoing and upcoming Expanded Access Protocols (EAPs) happening in parallel with the HEALEY ALS Platform Trial, and answered questions from the audience.
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November 30, 2023: Weekly Q&A
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital provided an overview of the HEALEY ALS Platform Trial to date, presented the weekly update, and answered questions from the audience.
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November 16, 2023: Regimen G Drug Science Q&A with Denali Therapeutics
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital, along with Regimen G co-lead Suma Babu, MBBS, MPH, and research scientists from Denali Therapeutics, presented the rationale and science behind DNL343, the investigational product being studied in Regimen G of the HEALEY ALS Platform Trial, and answered questions from the audience.
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November 9, 2023: Weekly Q&A and EAP Update with Suma Babu, MBBS, MPH
Suma Babu, MBBS, MPH of Massachusetts General Hospital presented news and updates about Expanded Access in the field of ALS research. Dr. Babu provided an overview of ongoing and upcoming Expanded Access protocols happening in parallel to the HEALEY ALS Platform Trial, and answered questions from the audience.
Watch Recording | Download Slides

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October 12, 2023: Weekly Q&A 
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented updates on the HEALEY ALS Platform Trial and answered questions from the audience. 
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September 28, 2023: Weekly Q&A and EAP Update
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented updates on the HEALEY ALS Platform Trial and answered questions from the audience. This week, the conversation focused on updates about Expanded Access progress and initiatives 
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September 21, 2023: Weekly Q&A with The ALS Association
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week's updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speakers Amanda Lee, MPH, MA and Sarah Parvanta, PhD, MPH from the ALS Association 
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September 14, 2023: Weekly Q&A with guest speaker Jen DiMartino 
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Jen DiMartino of ALS ONE. 
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September 7, 2023: Weekly Q&A
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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August 24, 2023: Weekly Q&A with guest speaker Robert Bowser, PhD
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented updates on the HEALEY ALS Platform Trial and answered questions from the audience. This week featured guest speaker Dr. Bob Bowser from Barrow Neurological Institute for a special discussion about his work advancing biomarker research in the field of ALS, and the importance of exploratory endpoints in ALS clinical trials. 
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August 17, 2023: Weekly Q&A with Gwen Petersen
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented updates on the HEALEY ALS Platform Trial and answered questions from the audience. This week featured guest speaker Gwen Petersen, an active ALS advocate and person living with ALS, to hear her perspective on research and helpful community resources. 
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August 10, 2023: Weekly Q&A featuring research participant
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented updates on the HEALEY ALS Platform Trial and answered questions from the audience. This week featured a research participant's story about his lumbar puncture experience, followed by a discussion with Dr. Senda Ajroud-Driss from Northwestern University and Dr. Shafeeq Ladha from Barrow Neurological Institute. 
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August 3, 2023: Weekly Q&A
Merit Cudkowicz, MD, MSc of Massachusetts General Hospital presented updates on the HEALEY ALS Platform Trial, provided an overview of ongoing regimens, and answered questions from the audience. 
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July 27, 2023: Weekly Q&A with guest speaker Suma Babu, MBBS, MPH
Suma Babu, MBBS, MPH of Massachusetts General Hospital hosted this week’s webinar about the HEALEY ALS Platform Trial and answered questions from the audience. Dr. Babu provided a brief overview of Expanded Access and presented an update about the Trehalose EAP specifically. 
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July 13, 2023: Weekly Q&A with guest speaker Sarah Heintzman, APRN-CNP
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by Sarah Heintzman, APRN-CNP, to discuss her role working on the Platform Trial at Ohio State University and to hear her perspective on the relationship between clinical care and research.
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July 6, 2023: Weekly Q&A with guest speaker Bill Cho, MD PhD
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented updates on the HEALEY ALS Platform Trial and answered questions from the audience. This week, we were joined by Bill Cho, MD PhD, of Calico Life Sciences to provide an overview of the science behind Regimen F and the investigational product ABBV-CLS-7262.
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June 29, 2023: Weekly Q&A with guest speaker Hilda Gutierrez, MD
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented updates on the HEALEY ALS Platform Trial and answered questions from the audience. This week, we were joined by Hilda Gutierrez, MD, research coordinator at Beth Israel Deaconess Medical Center in Boston. 
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June 22, 2023: Weekly Q&A with guest speaker Shafeeq Ladha, MD
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented updates on the HEALEY ALS Platform Trial. This week, we were joined by Dr. Shafeeq Ladha from Barrow Neurological Institute to share a step-by-step video guide about what to expect during a lumbar puncture, and discuss his perspective on the importance of CSF analysis in ALS research.
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June 15, 2023: Weekly Q&A
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented updates on the HEALEY ALS Platform Trial and answered questions from the audience. This week, we shared an overview of new online resources available for Regimen G.
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June 8, 2023: Weekly Q&A with guest speaker Brandy Quarles, MPH
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented updates on the HEALEY ALS Platform Trial and answered questions from the audience. This week, we were joined by guest speaker Brandy Quarles, MPH, Research Operations Coordinator at Augusta University in Georgia, to hear her perspective working on the Platform Trial. 
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June 1, 2023: Weekly Q&A
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented updates on the HEALEY ALS Platform Trial and answered questions from the audience. This week, Dr. Paganoni provided an overview of the Platform Trial to date and discussed future plans in the field of ALS research.
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May 25, 2023: Weekly Q&A
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented updates on the HEALEY ALS Platform Trial and answered questions from the audience. This week, Dr. Paganoni discussed the origin of the platform trial research design and reviewed Platform Trial progress to date.
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May 18, 2023: Weekly Q&A
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented updates on the HEALEY ALS Platform Trial and answered questions from the audience. This week, Dr. Paganoni discussed the evolving landscape of ALS treatments, reviewed currently approved medications for ALS, and explained the different mechanisms of action of study drugs included in the Platform Trial to date.

May 11, 2023: Weekly Q&A with Patient Navigation
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. Catherine Small, Patient Navigator for the trial, presented metrics collected over the past two and a half years since the Patient Navigator role initiated.
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May 4, 2023: Weekly Q&A
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. 
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April 27, 2023: Weekly Q&A with guest speaker Judi Carey, RN
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Judi Carey, RN (Research Access Nurse at the Healey Center for ALS) to discuss her role in clinical trials and her perspective on research.
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April 20, 2023: Weekly Q&A
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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April 13, 2023: Weekly Q&A
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week's updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by Dr. Shafeeq Ladha (co-lead investigator for Regimen E) and representative from Seelos Therapeutics to discuss recent news related to Regimen E.
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April 6, 2023: Weekly Q&A
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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March 30, 2023: Weekly Q&A with guest speaker James Berry, MD, MPH
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker James Berry, MD, MPH of Mass General to discuss cerebrospinal fluid (CSF) collection in ALS clinical trials and the lumbar puncture (LP) procedure. 
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March 23, 2023: Weekly Q&A with guest speaker Jeffrey Rothstein, MD, PhD
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Jeffrey Rothstein, MD, PhD, Director of the Robert Packard Center for ALS at Johns Hopkins University, to discuss the importance of identifying biomarkers in ALS, recent research findings related to TDP-43, and future directions in ALS research.
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March 16, 2023: Weekly Q&A with guest speaker Nicholas Maragakis, MD
Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Nicholas Maragakis, MD of Johns Hopkins Medicine to discuss recent research findings related to TDP-43, and the pivotal importance of exploratory biomarkers in ALS research. 
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March 9, 2023: Weekly Q&A and Regimen C Update
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by Regimen C co-Lead investigators, biostatisticians, and representatives from Clene Nanomedicine to discuss recent additional Platform Trial study results about CNM-Au8. The webinar was not recorded. Please submit any questions to healeyalsplatform@mgh.harvard.edu.

March 2, 2023: Weekly Q&A 
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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February 23, 2023: Weekly Q&A and Regimen D Update
Merit Cudkowicz, MD, MSc of Massachusetts General Hospital presented this week’s updates on the HEALEY ALS Platform Trial, discussed the topline results for Regimen D/pridopidine, and answered questions from the audience. We were joined by the co-lead investigators for Regimen D, several biostatisticians, and representatives from Prilenia Therapeutics. The webinar was not recorded, but another community webinar about the Regimen D results will be held on Monday, March 6th at 10am Eastern time.
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February 16, 2023: Weekly Q&A with guest speaker Jeremy Shefner, MD, PhD
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD of Massachusetts General Hospital presented this week’s updates on the HEALEY ALS Platform Trial, discussed the ways exploratory biomarkers in the Platform Trial are designed to advance science, and answered questions from the audience. We were joined by Jeremy Shefner, MD, PhD, a leading expert on outcomes and biomarkers in ALS research, from Barrow Neurological Institute in Phoenix, AZ. 
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February 9, 2023: Weekly Q&A
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial, discussed the ways exploratory endpoints and biomarkers in the Platform Trial are designed to advance science, and answered questions from the audience. 
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February 2, 2023: Weekly Q&A
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial, discussed the role of the Therapy Evaluation Committee in the selection of new regimens, and answered questions from the audience. 
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January 26, 2023: Weekly Q&A and Regimen C Update
Sabrina Paganoni, MD, PhD presented updates on the HEALEY ALS Platform Trial and answered questions from the audience. Guest speakers Michael Hotchkin (Chief Development Officer at Clene Nanomedicine, Inc) and James Berry, MD, MPH (Regimen C co-Lead, Mass General Hospital) joined the webinar again this week to recap the latest news and updates regarding Regimen C. 
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January 19, 2023: Weekly Q&A 
Sabrina Paganoni, MD, PhD presented this week's updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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January 12, 2023: Weekly Q&A and Regimen C Update
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speakers Michael Hotchkin (Chief Development Officer at Clene Nanomedicine, Inc) and James Berry, MD, MPH (Regimen C co-Lead, Mass General Hospital) to discuss the latest news and updates regarding Regimen C.
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January 5, 2023: Weekly Q&A 
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We looked back at Platform Trial progress since enrollment started in 2020 and discussed upcoming plans for the trial in 2023. 
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December 15, 2022: Weekly Q&A
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. 
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December 8, 2022: Weekly Q&A with guest speaker Mia Resendes
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Mia Resendes, Primary Research Coordinator for the Platform Trial at Mass General Hospital, to hear about her role in the trial and her experience working on Regimen E.
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December 1, 2022: Weekly Q&A with guest speaker Catherine Douthwright, PhD 
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Catherine Douthwright, PhD, Neuromuscular Clinical Research Program Director at UMass Chan Medical School in Worcester, to hear about her experience working on the Platform Trial.
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November 17, 2022: Weekly Q&A
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. The presentation reviewed resources to learn more about the currently enrolling Regimen E and provided a look forward at plans for Expanded Access in 2023.
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November 10, 2022: Weekly Q&A
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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October 27, 2022: Weekly Q&A
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. The presentation reviewed key aspects of the trial design and provided an update about the status of current regimens, a recap of recent research news, and a look forward at plans for 2023.
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October 20, 2022: Weekly Q&A with guest speaker Amanda Peltier MD, MS
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Amanda Peltier MD, MS, Site Investigator at Vanderbilt University in TN, to hear about her experience working in ALS research.
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October 13, 2022: Weekly Q&A 
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. The presentation provided an overview of Platform Trial progress to date and a look forward at plans for the addition of new regimens and grant-funded Expanded Access in 2023.
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October 6, 2022: Weekly Q&A featuring discussion of Regimen C/CNM-Au8 results
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by the co-lead investigators for Regimen C and representatives from Clene Nanomedicine, Inc. to discuss the trial results for CNM-Au8.
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September 29, 2022: Weekly Q&A featuring discussion of Regimen B/Verdiperstat results
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by the co-lead investigators for Regimen B and representatives from Biohaven Pharmaceuticals to discuss the trial results for Verdiperstat. 
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September 22, 2022: Weekly Q&A with guest speakers Meghan Hall and Mariah Connolly
Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speakers Meghan Hall, BS, CCRA, CCRP and Mariah Connolly, BS, CCRA to discuss the role of Site Monitors in the Platform Trial. 
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September 15, 2022: Weekly Q&A with guest speaker Tawfiq Al-Lahham, MD 
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Tawfiq Al-Lahham, MD, Site Investigator at UPMC in Pennsylvania.
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September 8, 2022: Weekly Q&A
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. 
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September 1, 2022: Weekly Q&A with guest host James Berry MD, MPH
James Berry MD, MPH of Mass General Hospital presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. The webinar featured a special discussion about exploratory endpoints and biomarkers. 
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August 25, 2022: Weekly Q&A with Guest Speaker Hani Kushlaf, MD, FAAN 
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Hani Kushlaf, MD, FAAN, Site Investigator at the University of Cincinnati in Ohio. 
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August 11, 2022: Weekly Q&A 
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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July 28, 2022: Weekly Q&A with guest biostatistician Lori Chibnik, PhD, MPH
The Patient Navigation team presented this week’s updates on the HEALEY ALS Platform Trial and welcomed return guest speaker Lori Chibnik, PhD, MPH. Dr. Chibnik led a discussion about the use of placebos in clinical trials and answered questions from the audience. 
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July 21, 2022: Weekly Q&A with guest speaker Allison Bulat
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Allison Bulat, Co-Chair of the NEALS Patient Education and Advocacy Committee, to learn about opportunities for patient advocacy in clinical research. 
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July 14, 2022: Weekly Q&A with guest speaker Eufrosina Young, MD
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Dr. Eufrosina Young, Platform Trial Site Investigator, SUNY Upstate, to learn about her role in the trial. 
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July 7, 2022 Weekly Q&A with guest speaker James Berry MD, MPH
Sabrina Paganoni, MD, PhD presented this week's updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker James Berry, MD, MPH for a special discussion about biomarkers.
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June 30, 2022: Weekly Q&A with guest speaker Gale Kittle, RN, MPH
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Gale Kittle, RN, MPH, Director of Outcome Measurements Training, to learn about her role in the trial.
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June 23, 2022: Weekly Q&A with guest speaker Jackie Whitesell, MD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Jackie Whitesell, MD, Site Investigator at Saint Alphonsus Regional Medical Center in Boise, ID.
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June 16, 2022: Weekly Q&A featuring Bruce Rosenblum, EAP Patient Advisory Committee Member
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Bruce Rosenblum, who currently serves as a member of the Patient Advisory Committee for the Platform Trial's companion Expanded Access Program (EAP).
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June 9, 2022: Weekly Q&A featuring Hospital for Special Care, CT
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speakers Honora Dalamagas, RN, MSN, CNS, and Natalie Cartwright, MS, from the Hospital for Special Care in New Britain, CT.
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June 2, 2022: Weekly Q&A with guest speaker Daniel Hevert, BS
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by Daniel Hevert, BS to discuss his perspective working as a Lead Clinical Research Coordinator on the trial.
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May 26, 2022: Weekly Q&A
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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May 19, 2022: Weekly Q&A with guest speaker Nadia Sethi, DDS
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by Nadia Sethi, DDS to discuss her perspective as a member of our Patient Advisory Committee.
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May 12, 2022: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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May 5, 2022: Weekly Q&A with guest speaker Peter Foss
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Peter Foss, President of ALS Finding a Cure.
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April 28, 2022: Weekly Q&A with guest speaker Kelly G. Gwathmey, MD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Kelly G. Gwathmey MD, Site Investigator at Virginia Commonwealth University in Richmond, VA.
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April 14, 2022: Weekly Q&A with guest speaker Kuldip Dave, PhD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Kuldip Dave, PhD, Vice President of Research at the ALS Association.
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March 31, 2022: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We shared new online resources about Expanded Access and discussed recent events in the field of ALS research.
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March 24, 2022: Weekly Q&A with guest speaker Jaimin Shah, MD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Jaimin Shah MD, Site Investigator at the Mayo Clinic in Jacksonville, FL.
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March 17, 2022: Weekly Q&A with guest speaker Edward J. Kasarskis, MD, PhD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Edward J. Kasarskis, MD, PhD, Site Investigator at the University of Kentucky in Lexington, KY, who discussed his research on FUS familial ALS and the importance of nutrition in ALS.
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March 10, 2022: Trehalose Mechanism of Action & Science
Sabrina Paganoni, MD, PhD scientists from Seelos Therapeutics, and regimen co-leads Merit Cudkowicz, MD, MSc and Shafeeq Ladha, MD presented the rationale and science behind trehalose, the investigational product being studied in Regimen E of the HEALEY ALS Platform Trial, and answered questions from the audience.
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March 3, 2022: Weekly Q&A with guest speaker Andrea Swenson, MD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Andrea Swenson, MD, Site Investigator at the University of Iowa in Iowa City, IA.
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February 24, 2022: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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February 17, 2022: Weekly Q&A with Guest Speaker Jim Caress, MD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Jim Caress, MD, Site Investigator at Wake Forest University in NC.
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February 10, 2022: Weekly Q&A with Guest Speaker Ximena Arcila-Londono, MD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial, shared estimated timelines for study results, and answered questions from the audience. We were joined by guest speaker Ximena Arcila-Londono, MD, Site Investigator at Henry Ford Hospital in Detroit, MI.
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February 3, 2022: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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January 27, 2022: Weekly Q&A
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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January 20, 2022: Weekly Q&A with guest speaker Senda Ajroud-Driss, MD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Senda Ajroud-Driss, MD, Site Investigator at Northwestern University in Chicago, IL.
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January 13, 2022: Weekly Q&A with guest speaker Sharon Hesterlee, PhD
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. We were joined by guest speaker Sharon Hesterlee, PhD, Chief Research Officer of the Muscular Dystrophy Association (MDA), to discuss the MDA's commitment to advancing ALS research.
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December 16, 2021: Weekly Q&A and discussion of biostatistics with guest Lori Chibnik, PhD, MPH
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial. She was joined by guest speaker Lori Chibnik, PhD, MPH to discuss biostatistics and answer questions from the audience.
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December 9, 2021: Weekly Q&A with guest speaker Nicholas Olney, MD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial, joined by guest speaker Nicholas Olney, MD, Platform Trial Site Investigator at the Providence ALS Center in Oregon, to discuss his experience working on the trial and answer questions from the audience.
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December 2, 2021: Weekly Q&A with guest speaker Neil Thakur, PhD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial. She was joined by guest speaker Neil Thakur, PhD, Chief Mission Officer of The ALS Association, to discuss the research pipeline and answer questions from the audience.
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November 18, 2021: Weekly Q&A with guest speaker James Berry, MD, MPH
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial. They were joined by guest speaker James Berry, MD, MPH to discuss biomarkers and answer questions from the audience.
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November 4, 2021: Weekly Q&A, Update from Clene Nanomedicine Inc., and Discussion of Biostatistics
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial. They were joined by guest speakers Robert Glanzman, MD, FAAN, and Michael Hotchkin of Clene Nanomedicine Inc., along with James Berry, MD, MPH and Nicholas Maragakis, MD, co-lead investigators for Regimen C of the Platform Trial. Lori Chibnik, PhD, MPH presented the role of biostatistics in clinical trials and answered questions from the audience.
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October 28, 2021: Weekly Q&A with Guest Speaker Megan Miller, PhD from I AM ALS
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience. They were joined by guest speaker Megan Miller, PhD, Director of Science and Policy at I AM ALS, to discuss the Expanded Access Protocol Program (EAP).
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October 21, 2021: Weekly Q&A with Guest Speaker Xiaowei (Bill) Su, MD, PhD from Penn State Health
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial with guest speaker Xiaowei (Bill) Su, MD, PhD from Penn State Health, and answered questions from the audience.
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October 14, 2021: Weekly Q&A with Guest Speaker David Walk, MD, FAAN from the University of Minnesota
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial with guest speaker, David Walk, MD, FAAN from the University of Minnesota and answered questions from the audience.
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September 30, 2021: Weekly Q&A with Guest Speaker Irfan Qureshi, MD from Biohaven Pharmaceuticals
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience with guest speaker Irfan Qureshi, MD from Biohaven Pharmaceuticals.
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September 23, 2021: Weekly Q&A with Guest Speaker Speaker Namita Goyal
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience with guest speaker Namita Goyal, MD from the University of California, Irvine.
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September 9, 2021: Weekly Q&A with Guest Speaker Jennifer DiMartino
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience with guest speaker Jennifer DiMartino, Executive Director of ALS ONE.
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September 2, 2021: Weekly Q&A with Guest Speaker Terry Heiman-Patterson, MD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial with guest speaker, Terry Heiman-Patterson, MD, from Temple University.
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August 26, 2021: Weekly Q&A with Guest Speaker Niraja Suresh, MD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial with guest speaker, Niraja Suresh, MD, from the University of South Florida .
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August 19, 2021: Weekly Q&A with guest speaker Laura Foster, MD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial with guest speaker, Laura Foster, MD, from the University of Colorado, Anschutz.
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August 12, 2021: Weekly Q&A with guest speaker, Michael Weiss, MD, FAAN
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial with guest speaker, Michael Weiss, MD, FAAN from the University of Washington.
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August 5, 2021: Weekly Q&A
Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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July 29, 2021: Weekly Q&A with guest speaker Hristelina Ilieva, MD, PhD 
James Berry MD, MPH presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience with guest speaker Hristelina Ilieva, MD, PhD from Thomas Jefferson University.
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July 22, 2021: Weekly Q&A with guest speaker Richard Bedlack, MD, PhD, MS
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial with guest speaker Richard Bedlack, MD, PhD, MS from Duke University.
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July 15, 2021: Weekly Q&A with guest speaker Shafeeq Ladha, MD
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial. They were joined by special guest Shafeeq Ladha, MD of the Barrow Neurological Institute.
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July 8, 2021: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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July 1, 2021: Weekly Q&A with guest speaker Jeffrey Rosenfeld, PhD, MD, FAAN
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience with guest speaker Jeffrey Rosenfeld, PhD, MD, FAAN of Loma Linda University.
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June 24, 2021: Weekly Q&A
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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June 17, 2021: Weekly Q&A with guest speaker Lauren Elman, MD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience with guest speaker Lauren Elman, MD from the University of Pennsylvania.
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June 10, 2021: Weekly Q&A
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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June 3, 2021: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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May 27, 2021: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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May 20, 2021: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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May 13, 2021: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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May 6, 2021: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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April 29, 2021: Weekly Q&A with guest speaker Eduardo Locatelli, MD
Sabrina Paganoni, MD, PhD presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience with guest speaker Eduardo Locatelli, MD from Holy Cross Hospital.
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April 22, 2021: Weekly Q&A with guest speaker Daragh Heitzman, MD, FAAN
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience, with guest speaker Daragh Heitzman, MD, FAAN of Texas Neurology.
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April 15, 2021: Weekly Q&A with guest speaker Adam Quick, MD
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial with guest speaker Adam Quick, MD, from The Ohio State University Wexner Medical Center and answered questions from the audience.
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April 8, 2021: Weekly Q&A with guest speaker Jeremey Shefner, MD, PhD
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial, discussed voice app biomarkers with guest speaker Jeremey Shefner, MD, PhD and answered questions from the audience.
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April 1, 2021: Weekly Q&A with guest speaker James Berry, MD, MPH
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial, discussed biomarkers with guest speaker James Berry, MD, MPH and answered questions from the audience.
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March 25, 2021: Weekly Q&A
Sabrina Paganoni, MD, PhD presented the week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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March 18, 2021: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented the week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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March 11, 2021: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented the week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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March 4, 2021: Weekly Q&A
Sabrina Paganoni, MD, PhD presented the week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience
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February 11, 2021: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented the week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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January 28, 2021: Weekly Q&A
Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD presented the week’s updates on the HEALEY ALS Platform Trial, discussed the trial design, and answered questions from the audience.
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January 14, 2021: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented the week's updates on the HEALEY ALS Platform Trial, discussed the Expanded Access Protocol (EAP) program, and answered questions from the audience.
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January 7, 2021: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented the week's updates on the HEALEY ALS Platform Trial, discussed the Expanded Access Protocol (EAP) program, and answered questions from the audience.
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December 17, 2020: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented the week’s updates on the HEALEY ALS Platform Trial, discussed open label extension and COVID impact, and answered questions from the audience.
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December 10, 2020: Weekly Q&A
Sabrina Paganoni, MD, PhD presented this week's updates on the HEALEY ALS Platform Trial, discussed the enrollment process and study visits, and answered questions from the audience.
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December 3, 2020: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented the week's updates on the HEALEY ALS Platform Trial and the Expanded Access Protocol (EAP) program, discussed the relationship between clinical research trials for ALS and clinical care, and answered questions from the audience.
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November 30, 2020: Monthly Update with guest speaker Jen DiMartino 
Merit Cudkowicz, MD, MSc, principal investigator, and Sabrina Paganoni, MD, co-principal investigator of the HEALEY ALS Platform Trial, provided an update on the progress of the HEALEY ALS Platform Trial. They were joined by Jen DiMartino of ALS One.
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November 19, 2020: Weekly Q&A
Sabrina Paganoni, MD, PhD presented the week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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November 12, 2020: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented the week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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November 5, 2020: Weekly Q&A
Sabrina Paganoni, MD, PhD presented the week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
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October 29, 2020: Monthly Update with guest speakers Troy Green and Peter Foss
Merit Cudkowicz, MD, MSc, principal investigator, and Sabrina Paganoni, MD, co-principal investigator of the HEALEY ALS Platform Trial, provided an update on the progress of the HEALEY ALS Platform Trial. They were joined by Troy Green from Tackle ALS and Peter Foss from ALS Finding a Cure.
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October 22, 2020: Weekly Q&A
Enrollment updates, trial sites, and patient questions.
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October 21, 2020: ALS/MND Alliance Clinical Trial Design Webinar
Merit Cudkowicz, MD, MSc joined researchers from Europe and Scotland to discuss new research and clinical trial models, how they will change the landscape going forward and their practical implications for patients and researchers.
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October 15, 2020: Weekly Q&A
Sabrina Paganoni, MD, PhD covers newly active trial sites, patient navigator service, and patient questions about the HEALEY ALS Platform Trial.
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October 13, 2020: CTTI Master Protocol Development Webinar for Researchers
Marianne Chase, the Senior Director Clinical Trial Operations at Mass General's Neurological Clinical Research Institute, joined representatives from the FDA, Janssen R&D and Edgewise Therapeutics to discuss the design of master protocol clinical trials, which allow researchers to answer multiple scientific questions within a single trial. Watch recording.

October 8, 2020: Weekly Q&A with guest speaker Sandy Morris
Mass General researchers Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD are joined by Sandy Morris of I AM ALS.
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September 29, 2020, 2020: Monthly Update with guest speaker Kuldip Dave, PhD
Merit Cudkowicz, MD, MSc, principal investigator, and Sabrina Paganoni, MD, co-principal investigator of the HEALEY ALS Platform Trial, provided an update on the progress of the HEALEY ALS Platform Trial. They were joined by Kuldip Dave, PhD, of the ALS Association.
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August 27, 2020, 2020: Monthly Update with guest speaker Taner Dagdelen
Sabrina Paganoni, MD, co-principal investigator of the HEALEY ALS Platform Trial, joined Taner Dagdelen, co-founder and CEO of Unite Genomics, for a webinar on August 27 to discuss the latest updates on the trial.
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July 7, 2020: Status Update
Healey Center faculty provide update on the status of the HEALEY ALS Platform Trial.
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April 8, 2020: Update and Message from Reagan Healey
We hosted a webinar on April 8th, 2020 to discuss the state of planning for the HEALEY ALS Platform Trial, featuring panelist Reagan Healey, who brought a message for us and for ALS patients and caregivers everywhere:

"I am really glad we could update you today and let you know of our neverending commitment you and your families. I am in New York and every night this month at 7 pm the city erupts with cheering, drum pounding and clapping to thank our Healthcare professionals and all frontline workers during this Corona Crisis. It can be heard as a low rumble if you’re inside and then a thunderous chorus when you open a window. But I am going to let you in on a secret: I scream and cheer for you all and for my brother Sean. And I want you to know that Sean and the Healey Center are and will continue to be the strongest voices on your behalf. We will put an end to the ALS crisis." – Regan Healey

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March 25, 2020: COVID Updates
As you all are, we too are closely monitoring the impact of COVID-19 on the HEALEY ALS Platform Trial timelines and operations. The bottom line is this: we are committed to beginning the trial as soon and as safely as we can.

January 22, 2020: Sean M. Healey & AMG Center for ALS at Mass General receives “May Proceed” notice for three drugs in first ALS platform trial
The Sean M. Healey & AMG Center for ALS at Mass General has received approval from the U.S. Food and Drug Administration (FDA) to proceed with administering three proposed drug regimens in the HEALEY ALS Platform Trial – the first trial of its kind for amyotrophic lateral sclerosis (ALS)

Merit Cudkowicz, MD, MSc, Director of the Healey Center for ALS, discusses the impact of the “May Proceed” notice from the FDA for three drugs in first ALS platform trial

January 14, 2020: ALS Association makes multi-year commitment to HEALEY ALS Platform Trial
The ALS Association announced a $3 million commitment to the first platform trial for ALS, taking place at the Sean M. Healey & AMG Center for ALS at Mass General in collaboration with the Barrow Neurological Institute and the Northeast ALS Consortium (NEALS).

September 18, 2019: Sean M. Healey & AMG Center for ALS at Mass General launches first ALS Platform Trial with 5 promising drugs
The Sean M. Healey & AMG Center at Massachusetts General Hospital will launch the first platform trial for amyotrophic lateral sclerosis (ALS) to accelerate the development of effective and breakthrough treatments for people with ALS.

Publications 

• Design and Statistical Innovations in a Platform Trial for Amyotrophic Lateral Sclerosis [May 2023] 

• Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development [December 2021]