Alice Melão,  —

Synpromics and Solid Biosciences have established a collaborative agreement to develop new potential gene therapies for the treatment of Duchenne muscular dystrophy (DMD). According to the partnership terms, Solid Biosciences will gain access to muscle-selective promoter candidates developed by Synpromics, which are…

A University of Utah medical professor has received a $600,000 Muscular Dystrophy Association grant to conduct a clinical trial aimed at understanding how congenital myotonic dystrophy develops over time. The three-year grant will allow Dr. Nicholas Johnson, an assistant professor of neurology, pediatrics, and pathology, to study what scientists call the natural history…

Sarepta Therapeutics says its lead candidate therapy for exon 53 skipping, golodirsen, showed potential to treat Duchenne muscular dystrophy (DMD) in a first clinical trial of DMD patients. According to results of the Phase 1/2 clinical study, 4053-101 (NCT02310906), golodirsen significantly boosted dystrophin protein production in 25 boys with confirmed deletions…

MRIs can pick up subtle muscle changes that standard tests cannot in slowly progressing disorders such as facioscapulohumeral muscular dystrophy, or FSHD, a study reports. The research in the journal PLoS ONE was titled “Long-term follow-up of MRI changes in thigh muscles of patients with Facioscapulohumeral dystrophy: A quantitative…

The first patient has been dosed in a Phase 2 clinical trial of ACE-083 as a treatment for the muscle impairment in Charcot-Marie-Tooth disease, according to the therapy’s developer, Acceleron Pharma. Acceleron is already evaluating ACE-083 in another Phase 2 trial — this one as a treatment for facioscapulohumeral muscular…

Researchers at the University of Missouri have developed a new method to efficiently deliver the correct form of dystrophin gene to muscles as a way to correct the faulty gene that characterizes Duchenne muscular dystrophy (DMD), a mouse study shows. Their study, “A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic…

Injecting a smaller but functional form of the dystrophin gene, called microdystrophin, into dogs naturally affected by Duchenne muscular dystrophy allowed them to recover muscle strength and stabilized their overall disease symptoms, a new study shows. “This preclinical study demonstrates the safety and efficacy of microdystrophin, and makes it possible to…

Sarepta Therapeutics has partnered with Idis Managed Access, a division of Clinigen Group, to develop a managed access program (MAP) for its lead therapy Exondys 51 (eteplirsen) for the treatment of Duchenne muscular dystrophy (DMD). The MAP was planned to provide early, expanded access to Sarepta’s Exondys 51…

The latest results of the Phase 3 clinical trial for PTC Therapeutics’ candidate Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) showed that it can benefit patients who are in a transition stage of the disease and are able to walk between 300 and…

The Muscular Dystrophy Association (MDA) has awarded Dr. Johanna Hamel, a neurologist at New York’s University of Rochester, a $130,000 fellowship to help her research the underlying mechanisms that cause myotonic dystrophy — the most common form of adult-onset muscular dystrophy. The American Academy of Neurology (AAN) and the American Brain Foundation…