muscular dystrophy
Muscular Dystrophy Association Announces First Project Under Kickstart Program
The first project in this program to advance gene therapies for very rare conditions will focus on CHAT-mutated congenital myasthenic syndrome.
Atamyo, Dion Foundation Expanding ATA-200 Muscular Dystrophy Trial to US
The foundation will fund the Phase Ib trial, which already has regulatory permission to proceed in Italy and France.
Genzyme Sues Sarepta for Alleged Patent Infringement on Muscular Dystrophy Treatment Elevidys
Genzyme has accused Sarepta of infringing two patents related to methods for preventing AAV aggregation and asked for unspecified damages.
FDA Converts Sarepta Therapeutics DMD Gene Therapy to Full Approval, Expands Indication
While some review teams had wanted to issue a complete response letter, CBER Director Peter Marks overruled the recommendation, per FDA documents.
Avidity Biosciences Expediting Development of AOC for Rare Muscular Dystrophy Therapy
Based on the benefits seen in facioscapulohumeral muscular dystrophy patients in a Phase I/II trial, the company said it is hoping to quickly launch registrational cohorts.