Treatment with SGT-001 — Solid Biosciences’ gene therapy candidate for Duchenne muscular dystrophy (DMD) — improves lung function, according to data from the first six patients enrolled in the ongoing IGNITE DMD clinical trial. The improvements, seen one year after a single infusion of the SGT-001 gene therapy into…
The European Commission (EC) has granted orphan designation to AOC 1001, Avidity Biosciences’ investigational therapy for myotonic dystrophy type 1 (DM1). This designation is given to investigational therapies that have the potential to treat rare, life-threatening, or very serious diseases that affect less than five in 10,000 patients…
Cumberland Pharmaceuticals is recruiting boys and men for a Phase 2 study evaluating the safety, efficacy, and duration of effect of its oral ifetroban therapy for cardiomyopathy — a disease of the heart muscle — linked to Duchenne muscular dystrophy (DMD). The study, called FIGHT DMD (NCT03340675),…
PepGen has raised funding totaling $112.5 million to advance toward the clinic its enhanced delivery oligonucleotide (EDO) lead programs in Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). The funding round keeps PepGen on track to start Phase 1 clinical trials in 2022 for DMD, and…
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