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

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISX9-CPC, IPS Heart ’s experimental stem cell ...

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

ROCKVILLE, MD, USA I June 5, 2025 I REGENXBIO Inc. (Nasdaq: RGNX) today announced new positive interim data from the Phase ...

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

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 ...

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

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

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

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...