Regenxbio Inc.’s gene therapy in treating Duchenne muscular dystrophy (DMD) produced positive initial phase I/II results from ...

REGENXBIO Inc. (Nasdaq: RGNX) today announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial. Updates ...

Gene therapies, exon-skipping drugs and next-gen steroids are reshaping Duchenne muscular dystrophy care, targeting broader ages and mutations. We are witnessing the fastest pivot in rare-disease ...

Regenxbio (RGNX) stock in focus as the company shares promising trial data for its RGX-202 gene therapy for Duchenne muscular ...

RegenXBio has shared updated phase 1/2 data on its Duchenne muscular dystrophy (DMD) gene therapy, providing evidence that ...

Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...

The Entertainment Software Association (ESA) today announced the 2025 recipients of the Interactive Entertainment Impact ...

A dad from Cumbria has raised over £5,500 for a muscular dystrophy charity after finishing the National Three Peaks Challenge ...

The Platform Technology Designation, which precedes the current FDA leadership, is designed to streamline the drug ...

The former workers cited various reasons why they think Novo was cautious: its conservative culture and history of investing ...

Leaders from the world of cell and gene therapy shared their personal stories and policy recommendations in an extraordinary roundtable conducted by the FDA in front of its recently-appointed top ...

European Commission approves Roche’s Evrysdi tablet to treat spinal muscular atrophy: Basel Thursday, June 5, 2025, 09:00 Hrs [IST] Roche announced that the European Commission ...