Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows ...

After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent ...

REGENXBIO (RGNX) reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE trial of ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

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

Most individuals diagnosed with a rare disease won’t live to become teenagers or adults. I knew what I had to do.

The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder ...

His mum also expressed the importance of highlighting muscular dystrophy, which predominantly affects boys and causes muscle loss in the thighs and pelvis, extending to the arms. Emily added ...

Newborns in Minnesota can now be screened for Duchenne muscular dystrophy (DMD) and guanidinoacetate methyltransferase (GAMT) deficiency. While there is no cure, treatment can make symptoms and ...

They say the truth is stranger than fiction, and seeing it brought into sharper focus through the lens of documentary filmmaking has given us some of our most unforgettable viewing experiences ...

About Duchenne Muscular Dystrophy DMD is a progressive neuromuscular disorder caused by a mutation in the DMD gene which affects the production of a protein called dystrophin. 3 Dystrophin is a ...

A new discovery about how tiny protein clusters form in cells could pave the way for treatments for Emery-Dreifuss muscular dystrophy (EDMD), a rare genetic disorder that causes muscle weakness ...