Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size. The ...

Clinical development and trial testing of vesleteplirsen (SRP-5051) for Duchenne muscular dystrophy (DMD) was stopped, with ...

But the first time I saw mine? At 43 years old. This was after three babies (my kids are 12, 12 and 8), and while working the ...

Muscle fibers in children with SMA type 1 show substantial differences in components of the myosin protein called heavy ...

In September, we shared 24-week dystrophin data from our FORWARD-53 trial, which included highly consistent mean muscle ...

The study demonstrates that activating the vitamin D receptor (VDR) with calcitriol suppresses cell proliferation in ...

Sarepta has stopped developing SRP-5051, its exon 51-skipping therapy for Duchenne MD, due to long-term safety concerns and ...

Nov. 12, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing ... including accelerated approval in the U.S. based on dystrophin ...

CAMBRIDGE, Mass., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology ...

Regular activity not only strengthens muscles but can bolster our bones, blood vessels, and immune system. MIT engineers have now found that exercise can also have benefits at the level of individual ...