US FDA Grants Expanded Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

Innovations in Care

We provide access to support and programs that empower the lives of people living with neuromuscular diseases.

Outside Organization Programs & Information

As part of our support to the neuromuscular disease community, MDA posts information provided by outside companies and organizations that are educational or research in nature.

The information, programs and resources listed below are not developed,  sponsored, funded or endorsed by MDA.  The companies and organizations posting below are responsible for all content and information provided. Any questions or queries, please email us at Posting may not be for fundraising or commercial purposes.

Clinical Trial Announcement for Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

PTC Therapeutics is announcing a study to evaluate the safety and pharmacokinetics of Ataluren in participants from ≥6 Months to <2 years of age with Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD). Information related to the clinical trial can be found here. If you need additional information or have questions, please contact PTC Therapeutics at or 1-866-562-4620.

LEMS Aware

LEMS Aware is a resource for patients who are seeking a diagnosis or resources for managing their LEMS. It was created to empower patients to advocate for themselves by educating them on symptoms of LEMS, searching fora LEMS specialist or even connecting them with other patients with Lambert-Eaton Myasthenic Syndrome. Wherever you are on your journey—searching for the right doctor, the right diagnosis, or the right treatment—this site can guide you.

Help ALS/IBM Research

Liberating Research, as part of a market research discussion, is conducting phone interviews and written diaries with people living with Inclusion Body Myositis (IBM) or Amyotrophic Lateral Sclerosis (ALS) and their partners/caregivers/loved ones to learn more about their experiences. For more information, please follow this link or contact the following: Emmanuel Konyani +442080048160 (UK)/ or Sharon Elliott (646) 701-0050 (US)/

PTC Therapeutics Online Events

PTC Therapeutics is a science-led, patient-centered biopharmaceutical company focused on discovering, developing and commercializing medicine for rare-disease patients. They offer free educational events throughout the year. The list of upcoming events, their descriptions, and RSVP information can be located on their Facebook page by following this link.

BIO is conducting a survey on the Impacts on COVID-Related Clinical Trial Alternations on Clinical Trial Participants.

This survey is being conducted by the Biotechnology Innovation Organization. We are hoping to hear from patients about how COVID-19 may have impacted or is currently impacting patients’ abilities to participate in clinical trials. Additionally, if trials have been altered, we are trying to better understand if the changes that have been implemented have reduced or added to the burden for patients participating in clinical trials. If you have any questions about this survey please contact Danielle Friend at We appreciate your participation! The survey results will be analyzed on a rolling bases beginning August 31, 2020. The questions outlined below apply only to experimental treatment trials, NOT natural history studies.


Background: For MG Awareness Month in partnership with patients we are excited to launch iMaGineMyMG. iMagineMyMG is a place for people living with and impacted by MG to listen, learn and connect with each other. Inspired and designed by people living with MG for people living with MG, iMaGineMyMG is a resource in learning more about MG. The site is filled with new ways to connect with others and share experiences, resources and information on life with MG. It includes opportunities to participate in monthly surveys, post videos and pictures and connect with others in the community. It also includes resources for individuals to talk with their doctors on improving their quality of life and the day to day experience of life with MG. We’ll be updating the site on a regular basis.

Exercise and CMT

The Charcot-Marie-Tooth Association in collaboration with Therapies for Inherited Neuropathies created a series of physical therapy centered videos to help CMT patients in the areas of flexibility, balance, strengthening, endurance and function.

No Charge Genetic Testing for Patients Suspected of Having SMA

The SMA Identified Program offers no charge genetic testing for patients suspected of having or clinically diagnosed with SMA.  The link to the program is below:

Take on Thymidine Kinase 2 deficiency (TK2d)

Whether you're living with an MDS, have been diagnosed with TK2d or are a parent, caregiver, or healthcare professional (HCP) who wants to learn more about MDS and TK2d, visit for more information. We can all take on TK2d together.

Gene Therapy Patient Education

The American Society of Gene and Cell Therapy has a variety of educational resources available on their website. Visit to learn more about the basics of gene therapy, the clinical trial process and the potential of gene therapy for specific diseases including SMA and MTM.