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

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 Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on ...

Investing.com -- Sarepta Therapeutics Inc (NASDAQ:SRPT) stock surged 11% Friday after the company announced plans to present 3-year topline functional results from its Phase 3 EMBARK study of ELEVIDYS ...

The father-of-two, Rich Clift, 38, will take on the 100km walk (spanning two days 16 - 17 May 2026), along with three others including brothers-in-law, Ashley ...

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, ...

The Healthcare Technology Report's Top 50 Healthcare Technology CEOs awards recognize executives whose ability to drive innovation, lead through change, and translate science into real-world ...

Hilary Swank found herself in an awkward and tense situation at a Los Angeles airport this week when she confronted a mother ...

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 ...