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

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

If you suffered losses exceeding $50,000 in Capricor between October 9, 2024 and July 10, 2025 and would like to discuss your ...

Shares of Avidity Biosciences, Inc. (NASDAQ:RNA) rose 4% Wednesday following the announcement that its drug candidate, ...

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

Precision BioSciences (NASDAQ:DTIL) saw its stock rise 5% following the announcement that the U.S. Food and Drug ...

A public workshop that columnist Shalom Lim and his girlfriend, Amanda, organized aimed to foster connection among people ...

BGENE-DMD is designed to treat Duchenne muscular dystrophy (DMD) via a one-time administration in a single AAV.

In the completed Phase 1/2 EXPLORE44 trial, del-zota demonstrated statistically significant increases in exon skipping, substantial increases in dystrophin production, and a significant reduction in ...

Investing.com -- Precision BioSciences (NASDAQ: DTIL) stock rose 5% after the company announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its PBGENE-DMD ...

Sarepta Therapeutics (SRPT) stock is back in focus as Roche (RHHBF) pauses global shipments of its Elevidys gene therapy ...

Discovered and developed by Sarepta Therapeutics, Elevidys is a gene therapy that won accelerated approval in June 2023 and ...