Myotonic dystrophies are inherited neuromuscular disorders characterized by progressive muscular wasting (dystrophy) and muscular stiffness (myotonia). It affects both muscles and others organs such as heart, eye, breathing appartus, endocrine system, digestive tract or nervous system, these are multisystem diseases.
Clinical trials on MD ongoing at the Institute:
- DYNE101-DM1-201: A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1
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