Genethon highlights latest developments in gene therapies for diseases once considered incurable, including Duchenne muscular dystrophy ...

A ladies' watch outshines the cost of many private jets. The Graff Diamonds Hallucination, a unique quartz timepiece ...

Shares of Sarepta Therapeutics gained after the company said it would present three-year, topline functional results from its Phase 3 study of a treatment for Duchenne muscular dystrophy. The stock ...

Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne ...

Nebraska families warn proposed Medicaid changes could force loved ones out of their homes and into institutions. Lawmakers ...

Researchers at Kumamoto University have demonstrated that iron supplementation can significantly alleviate muscle pathology and functional decline in a mouse model of facioscapulohumeral muscular ...

However, last year, two patients died from liver failure after receiving Elevidys. This led to Sarepta Therapeutics having to include a boxed warning for potential liver injury and liver failure, ...

He Jiankui’s name became synonymous with scientific transgression when his secret experiment produced the world’s first gene-edited babies. After serving a prison sentence in China, he is back in the ...

When it comes to children born with rare diseases, early diagnosis can make the difference between life and death. Since many ...

They're not looking at the bigger picture; they're just looking at dollar signs. I don't want to be labeled as a dollar sign.

The Brain Involvement iN Dystrophinopathies (BIND) screener is a robust tool that facilitates early identification of ...

FDA has requested the HOPE-3 clinical study report (CSR) as part of the BLA review processCompany expects to submit updates to the BLA in ...