Myotonic dystrophy type 1 (DM1) is a multi-systemic genetic disorder affecting the skeletal muscle, heart, brain, and other organs and is marked by progressive muscular weakness, atrophy and myotonia.
New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of individuals with Types 2 or 3 spinal muscular atrophy (SMA) Late-breaking ...
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