Below is a selection of research publications from the Healey Center for ALS at Massachusetts General Hospital. Learn more about our research programs or look up our full publication listing at PubMed.

Addressing Heterogeneity in ALS Clinical Trials.
Goyal NA, Berry JD, Windebank A, Nathan P, Maragakis NJ, van den Berg LH, Genge A, Miller R, Baloh RH, Kern R, Gothelf Y, Lebovits C, Cudkowicz M. Muscle & Nerve. 2020 Jan 3. doi: 10.1002/mus.26801. [Epub ahead of print] Review.
PMID: 31899540

NurOwn, phase 2, randomized, clinical trial in patients with ALS: Safety, clinical, and biomarker results.
Berry JD, Cudkowicz ME, Windebank AJ, Staff NP, Owegi M, Nicholson K, McKenna-Yasek D, Levy YS, Abramov N, Kaspi H, Mehra M, Aricha R, Gothelf Y, Brown RH. Neurology
. 2019 Nov 18. pii: 10.1212/WNL.0000000000008620. doi: 10.1212/WNL.0000000000008620. [Epub ahead of print]
PMID: 31740545

Selection design phase II trial of high dosages of tamoxifen and creatine in amyotrophic lateral sclerosis.
Babu S, Macklin EA, Jackson KE, Simpson E, Mahoney K, Yu H, Walker J, Simmons Z, David WS, Barkhaus PE, Simionescu L, Dimachkie MM, Pestronk A, Salameh JS, Weiss MD, Brooks BR, Schoenfeld D, Shefner J, Aggarwal S, Cudkowicz ME, Atassi N. Amyotroph Lateral Scler Frontotemporal Degener. 2019 Oct 14:1-9. doi: 10.1080/21678421.2019.1672750. [Epub ahead of print]
PMID: 31608711

Adjusted cost analysis of video televisits for the care of people with amyotrophic lateral sclerosis.
Paganoni S, van de Rijn M, Drake K, Burke K, Doyle M, Ellrodt AS, Nicholson K, Atassi N, de Marchi F, Babu S, Estrada J, Schwamm LH, Berry JD. Muscle Nerve. 2019 Aug;60(2):147-154. doi: 10.1002/mus.26606. Epub 2019 Jun 27.
PubMed PMID: 31136007.

Integration of a palliative care specialist in an amyotrophic lateral sclerosis clinic: Observations from one center.
Brizzi K, Paganoni S, Zehm A, De Marchi F, Berry JD. Muscle & Nerve. 2019 Aug;60(2):137-140. doi: 10.1002/mus.26607. Epub 2019 Jun 20.
PMID: 31172537.

Pharmacological Profiling of Purified Human Stem Cell-Derived and Primary Mouse Motor Neurons.
Moakley D, Koh J, Pereira JD, DuBreuil DM, Devlin AC, Berezovski E, Zhu K, Wainger BJ. Sci Rep. 2019 Jul 25;9(1):10835. doi: 10.1038/s41598-019-47203-7.
PMID: 31346219

Design and analysis of a clinical trial using previous trials as historical control.
Schoenfeld DA, Finkelstein DM, Macklin E, Zach N, Ennist DL, Taylor AA, Atassi N. Pooled Resource Open-Access ALS Clinical Trials Consortium. Clin Trials. 2019 Jul 1:1740774519858914. doi: 10.1177/1740774519858914. [Epub ahead of print]
PMID: 31256630

ALS/SURV: a modification of the CAFS statistic.
Goutman SA, Brown MB, Cudkowicz M, Atassi N, Feldman EL. Amyotroph Lateral Scler Frontotemporal Degener. 2019 Jul 23:1-8. doi: 10.1080/21678421.2019.1643375. [Epub ahead of print]
PMID: 31334681

All-Cause and Cause-Specific Mortality Among Major League Baseball Players.
Nguyen VT, Zafonte RD, Kponee-Shovein KZ, Paganoni S, Weisskopf MG. JAMA Intern Med. 2019 Jul 22. doi: 10.1001/jamainternmed.2019.1218. [Epub ahead of print]
PubMed PMID: 31329214; PubMed Central PMCID: PMC6646969.

Class I and II histone deacetylase expression is not altered in human amyotrophic lateral sclerosis: Neuropathological and positron emission tomography molecular neuroimaging evidence.
Dios AM, Babu S, Granucci EJ, Mueller KA, Mills AN, Alshikho MJ, Zürcher NR,Cernasov P, Gilbert TM, Glass JD, Berry JD, Atassi N, Hooker JM, Sadri-Vakili G.  Muscle & Nerve. 2019 Jun 26. doi: 10.1002/mus.26620. [Epub ahead of print]
PMID: 31241177.

When a negative trial in ALS has a positive effect on research.
Atassi N. Lancet Neurol. 2018 Aug;17(8):653-655. doi: 10.1016/S1474-4422(18)30210-2. Epub 2018 Jun 19.
PMID: 29934197

Nutritional counseling with or without mobile health technology: a randomized open-label standard-of-care-controlled trial in ALS.
Wills AM, Garry J, Hubbard J, Mezoian T, Breen CT, Ortiz-Miller C, Nalipinski P, Sullivan S, Berry JD, Cudkowicz M, Paganoni S, Chan J, Macklin EA. BMC Neurol. 2019 May 29;19(1):104. doi: 10.1186/s12883-019-1330-6.
PubMed PMID: 31142272; PubMed Central PMCID: PMC6540456.

VITALITY-ALS STUDY GROUP. A phase III trial of tirasemtiv as a potential treatment for amyotrophic lateral sclerosis.
Shefner JM, Cudkowicz ME, Hardiman O, Cockroft BM, Lee JH, Malik FI, Meng L, Rudnicki SA, Wolff AA, Andrews JA. Amyotroph Lateral Scler Frontotemporal Degener. 2019 May 13:1-11. doi:10.1080/21678421.2019.1612922. [Epub ahead of print] Erratum in: Amyotroph Lateral Scler Frontotemporal Degener. 2019 Jul 14;:1.
PMID: 31081694.

Provisional best practices guidelines for the evaluation of bulbar dysfunction in amyotrophic lateral sclerosis.
Contributing Members of the NEALS Bulbar Subcommittee.  Pattee GL, Plowman EK, Focht Garand KL, Costello J, Brooks BR, Berry JD, Smith RA, Atassi N, Chapin JL, Yunusova Y, McIlduff CE, Young E, Macklin EA, Locatelli ER, Silani V, Heitzman D, Wymer J, Goutman SA, Gelinas DF, Perry B, Nalipinski P, Stipancic K, O'Brien M, Sullivan SL, Pioro EP, Gargiulo G, Green JR. Muscle & Nerve. 2019 May;59(5):531-536. doi: 10.1002/mus.26408. Epub 2019 Jan 22.
PMID: 30620104.

Design and results of a smartphone-based digital phenotyping study to quantify ALS progression.
Berry JD, Paganoni S, Carlson K, Burke K, Weber H, Staples P, Salinas J, Chan J, Green JR, Connaghan K, Barback J, Onnela JP. Ann Clin Transl Neurol. 2019 Apr 3;6(5):873-881. doi: 10.1002/acn3.770. eCollection 2019 May.
PMID: 31139685

Disruption of RNA Metabolism in Neurological Diseases and Emerging Therapeutic Interventions.
Nussbacher JK, Tabet R, Yeo GW, Lagier-Tourenne C. Neuron. 2019 Apr 17;102(2):294-320. doi: 10.1016/j.neuron.2019.03.014. Review.
PMID: 30998900

Positron Emission Tomography Molecular Imaging Biomarkers for Amyotrophic Lateral Sclerosis.
Chew S, Atassi N.   Front Neurol. 2019 Mar 1;10:135. doi: 10.3389/fneur.2019.00135. eCollection 2019. Review.
PMID: 30881332

Lunasin does not slow ALS progression: results of an open-label, single-center, hybrid-virtual 12-month trial.
Bedlack RS, Wicks P, Vaughan T, Opie A, Blum R, Dios A, Sadri-Vakili G. Amyotroph Lateral Scler Frontotemporal Degener. 2019 Jan 21:1-9. doi: 10.1080/21678421.2018.1556698. [Epub ahead of print]
PMID: 30663902

Heterogeneity, urgency, generalizability, and enrollment: The HUGE balance in ALS trials.
Karam C, Berry JD.  Neurology. 2019 Jan 9. pii: 10.1212/WNL.0000000000006837. doi: 10.1212/WNL.0000000000006837. [Epub ahead of print]
PMID: 30626648

A pilot trial of RNS60 in amyotrophic lateral sclerosis.
Paganoni S, Alshikho MJ, Luppino S, Chan J, Pothier L, Schoenfeld D, Andres PL, Babu S, Zürcher NR, Loggia ML, Barry RL, Luotti S, Nardo G, Trolese MC, Pantalone S, Bendotti C, Bonetto V, De Marchi F, Rosen B, Hooker J, Cudkowicz M, Atassi N. Muscle & Nerve. 2019 Mar;59(3):303-308. doi: 10.1002/mus.26385. Epub 2018 Dec 26. PMID: 30458059 [PubMed - indexed for MEDLINE]

Telemedicine to innovate amyotrophic lateral sclerosis multidisciplinary care: The time has come.
Paganoni S, Simmons Z. Muscle & Nerve. 2019 Jan;59(1):3-5. doi: 10.1002/mus.26311. Epub 2018 Dec 18.
PMID: 30066337

Taking on the Elephant in the Tissue Culture Room: iPSC Modeling for Sporadic ALS.
Wainger BJ, Lagier-Tourenne C. Cell Stem Cell. 2018 Oct 4;23(4):466-467. doi: 10.1016/j.stem.2018.09.015.
PMID: 30290176

Animal models of neurodegenerative diseases.
Dawson TM, Golde TE, Lagier-Tourenne C. Nat Neurosci. 2018 Oct;21(10):1370-1379. doi: 10.1038/s41593-018-0236-8. Epub 2018 Sep 24. Review.
PMID: 30250265

Integrated imaging of [11C]-PBR28 PET, MR diffusion and magnetic resonance spectroscopy 1H-MRS in amyotrophic lateral sclerosis.
Ratai EM, Alshikho MJ, Zürcher NR, Loggia ML, Cebulla CL, Cernasov P, Reynolds B, Fish J, Seth R, Babu S, Paganoni S, Hooker JM, Atassi N. Neuroimage Clin. 2018 Aug 9;20:357-364. doi: 10.1016/j.nicl.2018.08.007. eCollection 2018.
PMID: 30112276

Integrated magnetic resonance imaging and [11 C]-PBR28 positron emission tomographic imaging in amyotrophic lateral sclerosis.
Alshikho MJ, Zürcher NR, Loggia ML, Cernasov P, Reynolds B, Pijanowski O, Chonde DB, Izquierdo Garcia D, Mainero C, Catana C, Chan J, Babu S, Paganoni S, Hooker JM, Atassi N.  Ann Neurol. 2018 Jun;83(6):1186-1197. doi: 10.1002/ana.25251.
PMID: 29740862

Nonmotor manifestations in ALS patients with tracheostomy and invasive ventilation.
Atassi N.  Muscle & Nerve. 2018 May;57(5):699-700. doi: 10.1002/mus.26068. Epub 2018 Feb 19. No abstract available.
PMID: 29342311

Exploring the Use of Educational Material About Shoulder Dysfunction: A Quality Improvement Project in People With Amyotrophic Lateral Sclerosis.
Burke K, Ellrodt AS, Levine J, Adams T, Allis R, Macmurdie I, Paganoni S.Am J Phys Med Rehabil. 2018 May;97(5):379-382. doi: 10.1097/PHM.0000000000000885.
PMID: 29303793

Urate levels predict survival in amyotrophic lateral sclerosis: Analysis of the expanded Pooled Resource Open-Access ALS clinical trials database.
Paganoni S, Nicholson K, Chan J, Shui A, Schoenfeld D, Sherman A, Berry J, Cudkowicz M, Atassi N. Pooled Resource Open-Access ALS Clinical Trials Consortium. Muscle & Nerve. 2018 Mar;57(3):430-434. doi: 10.1002/mus.25950. Epub 2017 Sep 21.
PMID: 28857199