Synpromics and Solid Biosciences have established a collaborative agreement to develop new…
Articles by Alice Melão
A University of Utah medical professor has received a $600,000 Muscular Dystrophy Association grant to conduct a clinical trial aimed at understanding…
Sarepta Therapeutics says its lead candidate therapy for exon 53 skipping, golodirsen, showed potential to treat Duchenne muscular dystrophy (DMD) in…
MRIs can pick up subtle muscle changes that standard tests cannot in slowly progressing disorders such as facioscapulohumeral muscular…
The first patient has been dosed in a Phase 2 clinical trial of ACE-083 as a treatment for the muscle…
Researchers at the University of Missouri have developed a new method to efficiently deliver the correct form of dystrophin gene…
Injecting a smaller but functional form of the dystrophin gene, called microdystrophin, into dogs naturally affected by Duchenne muscular dystrophy allowed…
Sarepta Therapeutics has partnered with Idis Managed Access, a division of Clinigen Group, to develop a managed access program…
The latest results of the Phase 3 clinical trial for PTC Therapeutics’ candidate Translarna (ataluren) for the treatment…
The Muscular Dystrophy Association (MDA) has awarded Dr. Johanna Hamel, a neurologist at New York’s University of Rochester, a $130,000 fellowship…