Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...

Sarepta will halt all shipments of Elevidys, its drug for Duchenne muscular dystrophy patients, by Tuesday evening.

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO ...

The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

After decades of research, gene therapy has been approved for Duchenne Muscular Dystrophy, a degenerative muscle disease diagnosed each year in about 1 in 3,500 newborns, almost all boys.

Managing osteoporosis and bone health in Duchenne muscular dystrophy occurs in a high-risk environment for fractures due to ...

2025年7月18日，FDA发布声明，要求Sarepta立即自愿停止Elevidys的全部商业供应。此前公司已主动停止对非行走患者的供药，但依然持续为尚能行走的患儿供药。面对FDA的请求，Sarepta选择拒绝，理由是“行走人群中并无新增或变化的安全 ...

Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...