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 ...

For example, there is an 11-year-old boy with Duchenne muscular dystrophy, which causes progressive muscle degeneration. He ...

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 ...

REGENXBIO stock is trading higher on Monday after the company reached a critical point in its trial for Duchenne gene therapy.

Regenxbio shares surged 20% after disclosing new, positive efficacy and safety data of its Duchenne muscular dystrophy treatment. Shares were recently trading around $11.55. The stock is down 46% on ...