Sarepta Therapeutics‘ microdystrophin gene therapy enhanced dystrophin protein expression in the muscles of four boys with Duchenne muscular…
Articles by Patricia Inácio, PhD
The U.S. Food and Drug Administration has lifted the clinical hold on a Phase 1/2 trial evaluating Sarepta Therapeutics‘ micro-dystrophin …
Two new research projects have been funded by the Coalition to Cure Calpain 3 (C3), a nonprofit patient advocacy…
PTC Therapeutics announced the winners of the 2018 global Strategies to Realize Innovation, Vision and Empowerment…
A global Phase 3 trial evaluating the efficacy and safety of oral edasalonexent in patients with Duchenne muscular dystrophy (DMD) will…
Summit Therapeutics is stopping the clinical development of ezutromid, which it was investigating as a Duchenne muscular dystrophy (DMD)…
The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on the Phase 1/2 trial testing SGT-001, a dystrophin…
The Centers for Disease Control and Prevention (CDC) released new, updated guidelines for the management of…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to GBC0905 as a potential treatment for facioscapulohumeral muscular…
The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines Agency (EMA) — is recommending against …