MP1032, MetrioPharm’s oral therapy candidate for Duchenne muscular dystrophy (DMD), has been designated an orphan drug by the U.S.
Articles by Patricia Inácio, PhD
Dyne Therapeutics’ investigational therapy DYNE-101 has been granted an orphan drug designation by the European Medicines Agency (EMA) for…
AOC 1044, an investigational exon 44-skipping therapy for Duchenne muscular dystrophy (DMD), has been granted fast track status by…
Having reached an agreement with the U.S. Food and Drug Administration (FDA), NS Pharma will be launching a Phase…
In a bid to further understand and solve potential gene therapy safety concerns in Duchenne muscular dystrophy (DMD), the…
AOC 1020, Avidity Biosciences’ investigational therapy for facioscapulohumeral muscular dystrophy (FSHD), has been granted orphan drug status by the…
PGN-EDODM1, a therapy candidate for myotonic dystrophy type 1 (DM1), showed a favorable safety profile in preclinical studies, including those…
Sarepta Therapeutics‘ investigational GALGT2 gene therapy helped stabilize muscle function when delivered at a higher dose to a boy…
EDG-5506, Edgewise Therapeutics’ experimental oral therapy, continues to safely reduce muscle damage and improve physical function in adults with…
Dystrogen Therapeutics’ experimental cell therapy, DT-DEC01, continues to show signs of efficacy in three boys with Duchenne muscular…