The FDA expanded the indication of eculizumab (Soliris) to include pediatric patients ages 6 and older with generalized ...

Sarepta Therapeutics said on Tuesday that a 16-year-old boy died from acute liver failure months after receiving the company's U.S-approved gene therapy for a rare muscular dystrophy. The patient ...

Chronic inflammatory demyelinating polyneuropathy (CIPD) can have a substantial impact on patients everyday life and, ...

The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but ...

Sarepta Therapeutics shares dropped 20% after a patient's death raises Elevidys safety concerns. Read why I am downgrading ...

Sarepta Therapeutics Inc.'s stock tumbled 23% on Tuesday after the biotech said a patient who used its gene therapy Elevidys for the rare disease Duchenne muscular dystrophy has died of acute liver ...

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...

The Food and Drug Administration has accepted Capricor Therapeutics' application seeking approval of its cell therapy deramiocel for muscular dystrophy patients and granted it a priority review.

Aside from muscular dystrophy, other examples of rare diseases include hemophilia, an inherited blood disorder that causes excessive bleeding and bruising; phenylketonuria, an inherited disorder ...

Preventive treatment with standard heart medications — when given before the onset of cardiac troubles — extends survival among males with Duchenne muscular dystrophy (DMD), according to data from the ...

Jang Ik-sun, a 37-year-old student from South Korea, has achieved an extraordinary academic milestone despite living with muscular dystrophy, a condition that has left him almost entirely ...