Heart problems can progress quickly in people with myotonic dystrophy type 2 (MD2) or facioscapulohumeral muscular dystrophy type 1 (FSHD1), even in…
Articles by Margarida Maia, PhD
Treatment with SGT-001 — Solid Biosciences’ gene therapy candidate for Duchenne muscular dystrophy (DMD) — improves lung function, according…
The European Commission (EC) has granted orphan designation to AOC 1001, Avidity Biosciences’ investigational therapy for myotonic dystrophy…
Cumberland Pharmaceuticals is recruiting boys and men for a Phase 2 study evaluating the safety, efficacy, and duration of…
PepGen has raised funding totaling $112.5 million to advance toward the clinic its enhanced delivery oligonucleotide (EDO) lead programs in…