Explore This Center


The mission of the CRND is to create new opportunities to improve the lives of those affected by rare, monogenic neurological disorders by building new alliances based on insights from biology, clinical unmet needs, and recent advantages in technology. Throughout strategic decision making, the patient perspective guides our process.

The vision of the CRND is a paradigm shift in research and treatment of rare diseases. We endeavor to empower patient populations previously isolated by their rare diseases. Through mentorship, collaboration, and targeted research, the CRND facilitates early proof of concept studies and clinical trials. Together, we are striving to improve the lives of those affected by understudied and newly discovered neurological disorders.​

We achieve this through:

  • Advancement of research through biological insights conducted at the Center for Genomic Medicine
  • Providing patient care through multidisciplinary clinics
  • Mentorship of young investigators engaging in rare diseases
  • Commitment to translational research, from bench to bedside
  • Exploration of partnerships and collaborations with academia, patients, and industry

In an effort to bring together rare disease experts from across departments, hospitals, and private industry, the CRND participates in quarterly “Think Tank” meetings sponsored by the Mass General Division of Clinical Research. These strategic discussions give clinical researchers the opportunity to share developments and collaborate with companies invested in the rare disease space.

Our Team

The CRND is committed to providing excellent clinical care and access to a variety of resources such as groundbreaking research opportunities and novel therapies. To accomplish this, our team consists of physicians, clinicians, researchers, and coordinators with a variety of specialties and interests. Many of our physicians are national and international leaders in the rare disease community and have specialized expertise in the diagnosis and treatment of a wide range of rare neurological disorders.


The goal of the CRND is to discover new treatments for rare diseases through translational research conducted at the Center for Genomic Medicine. Unlike traditional approaches in academic research, we focus on early identification of knowledge gaps, allowing for a streamlined drug development and trial implementation. We participate in many ongoing research initiatives from bench to bedside.

Clinical Programs

The CRND takes a multidisciplinary approach to providing excellence in patient-centered care. We hold several specialized clinics, bringing together a collaborative care team with many different experiences and perspectives. Our patients have access to a team of specialists, accurate diagnoses, personalized treatments, and cutting-edge research opportunities.

Patient Resources

We understand that it can be difficult for people with rare diseases to understand their condition, the medical issues that they may encounter, and ways to manage their symptoms. In addition to partnering with many patient advocacy groups whose missions are to improve the lives of patients living with such diseases, we participate in multiple research initiatives aimed at treating rare neurological diseases and collaborate with several clinical programs specialized in diagnosing and treating such diseases.

Read our newsletters:

February Newsletter

November Newsletter


One of the ways we achieve our goal of advancing treatment of rare diseases and empowering the rare disease community is through partnerships and collaborations with academia, patient advocacy groups, and industry.​ For more information on how to collaborate with the CRND, contact us at 617-724-1379 or crnd@partners.org.

Industry Partnerships

Private industry holds crucial knowledge on drug development and regulatory processes. The close proximity of MGH to many innovative companies with a shared interest in scientific discovery provides an ideal opportunity for partnership. By de-risking early phase development for rare disease therapies, the CRND makes these conditions attractive for industry engagement and investment. Teaming with Partners HealthCare Innovation helps the CRND work in concert with private industry.

Clinical trials wouldn't be possible without our sponsors:

Patient Advocacy Partnerships​

Patient engagement is essential for drug development in orphan diseases. We seek to overcome the hierarchies of conventional patient-doctor relationships, encouraging mutual listening and sharing of knowledge and insight within the clinic.

We currently partner with many patient advocacy groups, including: