Author Archives: Margarida Maia PhD

Changes to Heart Can Be Evident in Myotonic Dystrophy Type 2, FSHD1

Heart problems can progress quickly in people with myotonic dystrophy type 2 (MD2) or facioscapulohumeral muscular dystrophy type 1 (FSHD1), even in those showing no symptoms of cardiac disease, a study reported. “MD2 and FSHD1 patients should be carefully followed-up to identify early development of remodeling and potential risks for the development…

Gene Therapy SGT-001 Improves Lung Function in DMD Boys in Trial

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…

AOC 1001 Earns Orphan Drug Status for DM1 in Europe

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…