An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...

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 ...

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

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 ...

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

The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...

The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

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 ...

On Thursday 28 August, Sam Taylor, 46, his wife Sue, 47, and daughter Beth, 17, from Crooklands in Cumbria will set off to ...