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

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

Duchenne muscular dystrophy (DMD) has long been one of the most devastating genetic disorders to affect children. Marked by progressive muscle degeneration, early loss of mobility, and eventual ...

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 ...

ELEVIDYS is approved for individuals ages 3- to less than 8-years-old, who do not have any deletions in exon ... in advancing the treatment of Duchenne muscular dystrophy through innovative ...

Certain genetic mutations in patients with DMD influence the timing of ambulation loss, with exon 44 skipping ... phenotype correlations in Duchenne muscular dystrophy (DMD).

Duchenne muscular dystrophy has seen more progress in the ... This includes questions about the ability to combine gene therapy with exon-skipping treatments for those patients that are amenable.

Japanese drugmaker Nippon Shinyaku said on Monday that its Duchenne muscular dystrophy drug ... belongs to a contentious class of drugs called exon-skippers. Duchenne is caused by mutations ...

Opens in a new tab or window DENVER -- Viltolarsen (Viltepso) was well tolerated by boys and men with Duchenne muscular dystrophy (DMD ... function with the exon-skipping therapy, as measured ...

An open-label extension study for patients with Duchenne muscular dystrophy who participated in studies of SRP-5051 (vesleteplirsen). ClinicalTrials.gov. Updated September 28, 2023.