The FDA cleared Sarepta to start dosing in a first-in-human clinical trial testing the gene therapy SRP-9005 in people with LGMD type 2C.

2,3 which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac ...

Columnist Shalom Lim celebrates two years of his relationship with reflections on life, love, and his experience with ...

Entering the age of 2 years, almost most children will ... However, until the 1980s, little was known about the causes of any type of muscular dystrophy. In 1986, researchers supported by the ...

A recent discovery of a molecular connection between autism and myotonic dystrophy, a type of neuromuscular disease, may provide a breakthrough on how clinicians approach autism spectrum disorder.

today shared updates from its clinical programs focused on limb-girdle muscular dystrophy (LGMD) subtypes 2C/R5, 2D/R3, and 2E/R4. SRP-9005 for LGMD type 2C/R5: Following input from the U.S. Food ...