Sarepta Therapeutics (SRPT) added ~25% in the morning hours on Wednesday after the company posted initial data from Phase 1/2 ...

Sarepta stock popped Wednesday after the company reported promising results for its gene-silencing tech in two forms of muscular dystrophy.

Sarepta (SRPT) stock rises on positive Phase 1/2 siRNA trial data showing muscle delivery, biomarker activity, and safety in ...

When most people think about music venues, gas stations do not come to mind. But the organizers of the Muscular Dystrophy ...

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

Muscle tissue damage appears when muscular dystrophy is induced in a mouse model (middle). But when researchers block the function of two genes that drive unwanted mitochondrial pore formation (right) ...

While it’s established that Duchenne muscular dystrophy (DMD) causes a loss of muscle mass and strength, few studies have examined the gastrointestinal issues tied to DMD. Initial fractures are the ...

Vinny DeMando, 8, has been experiencing rapid mobility changes among rare disease progression. The community has rallied ...

The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...

A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. The creation of the ...

Muscular Dystrophies are a group of genetic conditions characterized by muscle weaknesses. They are genetic conditions and have different sub-categories depending on which area of muscle mass is ...

Longboat Key firefighters “filled the boot” through a fundraising effort for the Muscular Dystrophy Association.