We need a dedicated framework that clarifies the rules, requirements and incentives for those building personalized ...

The Swiss company, which markets Elevidys abroad, said it believes the benefit-risk balance to treatment remains positive in ...

Researchers led by UC Davis Health scientist Sanchita Bhatnagar have developed a promising gene therapy that could treat Rett syndrome. The therapy ...

In this week’s edition of InnovationRx, we look at Moderna’s use of quantum computers, a new top drug regulator at the FDA, ...

The Purdy family shares their insights on navigating their child’s rare neurodegenerative disease and their direct ...

Q2 2025 has demonstrated the pharmaceutical industry’s continued momentum in developing transformative therapies across ...

Roche is pausing shipments of Elevidys in some countries, following partner Sarepta Therapeutics’ move in the U.S., as safety ...

Radiation can induce amphiregulin expression in tumor cells, increasing their size and shortening patient survival.

Sarepta faces an “arduous and treacherous path” to try to get its Duchenne muscular dystrophy therapy Elevidys back onto the ...

Solid Biosciences said federal regulators have granted its fast-track designation for the company's investigational gene therapy for the treatment of a heart rhythm condition.

Shares of Cambridge, Mass.-based Sarepta have fallen by more than 90% in the last year amid safety concerns about its Elevidys, a gene therapy that treats Duchenne muscular dystrophy, following ...

Get the 2025 update on the biotech companies Labiotech spotlighted in 2024 — who advanced, who stalled, and who surprised us.