Myotonic Dystrophy Research
Although myotonic dystrophy (DM) currently has no treatment or cure, our mission at Myotonic is to increase the knowledge and science available to any researcher or clinician seeking to uncover answers. We also continually aim to attract new researchers to the DM field by awarding grants to those focused on molecular biology and basic science in DM research, as well as the care, treatment and support of those living with DM.
Myotonic recently executed a $5 million, 3-year drug development acceleration effort to attract more industry investment, lower barriers to therapy development, and expand the amount of data available to drive discovery. We fund research fellows, clinical trial infrastructure projects and other key research, and maintain one of the largest DM patient registries in the world.
We have significantly improved care for people living with myotonic dystrophy by developing the first-ever international clinical care recommendations for DM1, DM2, congenital DM, cardiology and pulmonology.
We’ve worked with the U.S. Social Security Administration to improve access to disability benefits for people living with DM and had congenital DM classified under its Compassionate Allowance program.
Myotonic successfully advocates for additional DM funding at several federal agencies and works with regulatory agencies to optimize the drug review approval process.
Click here to read the latest medical and research articles in the News and Updates section on the website.
Click here to learn about recipients of research grants and their innovative DM research.
Help Our Researchers through Registering
The Myotonic Dystrophy Family Registry (MDFR) helps academic, biotech, pharma and other researchers to develop new, effective treatments for myotonic dystrophy. When you join the Registry, researchers and other professionals can access de-identified aggregate data to identify target populations for study planning or recruitment for research studies and clinical trials.
Registering your profile also provides access to explore the database and use the Registry’s data analysis and report capabilities to learn more about DM and the community of people living with it. Click here to go to the Registry website, learn more and sign up.