Dossier
Sarepta Therapeutics
Coverage of Sarepta Therapeutics in the Nexus archive.
- STAT+: Regenxbio says Duchenne gene therapy succeeded in clinical trial, paving way for FDA submission
Regenxbio's experimental gene therapy for Duchenne muscular dystrophy has produced high levels of a miniaturized muscle protein, paving the way for FDA submission. The company aims to create a safer and more effective therapy than Sarepta Therapeutics' Elevidys. Regenxbio is seeking accelerated approval.
- STAT+: Next-gen Duchenne drug from Entrada disappoints
Entrada Therapeutics' next-generation drug for Duchenne muscular dystrophy disappointed in an early trial, raising questions about the company's competitiveness. The drug is designed to help patients produce shortened but still functional forms of dystrophin. Scientists have devised ways to redesign these molecules for better results.