A global Phase 3 trial evaluating the efficacy and safety of oral edasalonexent in patients with Duchenne muscular dystrophy (DMD) will…
Articles by Patricia Inácio, PhD
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 …
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to investigational therapy Sarconeos for…
Exondys 51 (eteplirsen), an RNA-targeted therapy marketed by Sarepta Therapeutics, is the first approved treatment…
The investigational therapy edasalonexent was seen to slow disease progression in boys with Duchenne muscular dystrophy, almost one-year results from…
Magnetic nanotubes with a spear-like tip may enhance the precision and effectiveness of gene therapy delivery, a promising therapeutic strategy…