Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne, commemorated its 30th ...

Columnist Shalom Lim tells an international conference about his experiences with inclusion during his education in Singapore ...

Milestone represents a significant step forward in the development of deramiocel for the treatment of Duchenne muscular ...

Trump fingers Robert F. Kennedy Jr. to lead the HHS, lupus and ATTR-CM dominate headlines this week, bluebird bio has a cash gap to leap and RegenxBio eyes Sarepta in Duchenne muscular dystrophy.

Oxford-based SynaptixBio, which is developing an antisense oligonucleotide (ASO) to ‘silence’ the expression of a single mutated gene, says the ASO market is set to grow dramatically, largely because ...

Disabled people give testimony in phase three of the Covid inquiry. The latest part of the Covid inquiry, which looks at the impact of the pandemic on the NHS, has heard powerful evidence from ...

Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy ...

The company expects the trial to support the submission of a biologics license application with the US Food and Drug Administration in 2026.

Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for ...

Elevidys, a gene therapy from Sarepta Therapeutics (SRPT.O), opens new tab approved in the United States last year for ...

Regenxbio’s investigational gene therapy improved muscle function for patients with Duchenne muscular dystrophy (DMD) in a phase 1/2 study. | Regenxbio’s investigational gene therapy improved muscle ...