To develop new therapies for patients living with myotonic dystrophy (DM), specific infrastructure and information needs to be in place to make clinical trials feasible. The information needed for CDM trials is likely to be different than that used for adult trials.
Marsha Dugan likes to describe herself as simply a stay-at-home mom, and avoids talking about her MBA and experience in international business, finance and marketing. Marsha, 60, who stepped back from the business world to raise her two sons, joined the board of the Myotonic Dystrophy Foundation in January 2017.
After a year of outreach and education, MDF has successfully convinced the Social Security Administration (SSA) to list congenital myotonic dystrophy (CDM) in its Compassionate Allowances program, significantly easing the application and review process.
AMO Pharma released interim results for a Phase 2a safety and toxicity study of Tideglusib (also known as AMO-02) for adolescents and adults with congenital myotonic dystrophy.
New data is available on the frequency, progression and treatment of GI symptoms in DM1 and DM2.
A new review article assesses the current status of and recommends future directions for brain imaging studies in DM1.
Given the brain manifestations of DM1, how aware are most patients of the impact and progression of their disease?
Learn more about Dr. Ami Mankodi, principal investigator at the National Institutes of Health’s (NIH) National Institute of Neurological Disorders and Stroke (NINDS) in Bethesda, Maryland. Dr. Mankodi has been involved in research that has helped shape a fundamental biologic and molecular understanding of myotonic dystrophy (DM).
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