The first patients with Duchenne muscular dystrophy (DMD) have been enrolled into the U.K.’s Early Access to Medicines Scheme (EAMS) for Santhera Pharmaceuticals’ Raxone (idebenone), giving patients access to the treatment before its regulatory approval. Raxone was included in EAMS after the Medicines and Healthcare Products Regulatory Agency (MHRA)…
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Sarepta’s Exon 53 Skipping Therapy, Golodirsen, Improves Dystrophin Expression in Phase 1/2 Trial
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…
PTC Therapeutics recently announced the recipients of the 2017 global Strategies to Realize Innovation, Vision and Empowerment (STRIVE) Awards program, recognizing excellence and innovation in the Duchenne muscular dystrophy (DMD) community. The 2017 awards were on World Duchenne Awareness Day, on Sept. 7. The STRIVE program was launched…
Parent Project Muscular Dystrophy is holding an awareness, fund-raising and educational campaign throughout September to fight Duchenne muscular dystrophy (DMD). It has scheduled events every day of Duchenne Action Month. You’ll find a calendar of activities here. September is traditionally a busy month for the worldwide DMD community.
A single gene therapy that silences the mutation responsible for oculopharyngeal muscular dystrophy (OPMD) and replaces the mutated gene with a normal one may advance into human studies in the second half of 2018. Benitec Biopharma started its OPMD program in 2014 and now announced their clinical candidate BB-301…
Parent Project Muscular Dystrophy (PPMD) will host a one-hour webinar at 2 p.m. Eastern Time on Wednesday, Sept. 6, that will focus on an upcoming clinical trial exploring gene therapy for Duchenne muscular dystrophy. The webinar will be led by Dr. Jerry Mendell, who, together with fellow researcher Dr. Louise…
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Aberration in RNA Processing Leads to Development of Congenital Myotonic Dystrophy, Study Finds
In what could be the first step on the long road to finding a potential cure, researchers have found that aberrations in RNA processing are the major cause of congenital myotonic dystrophy (CDM). The study “Disrupted prenatal RNA processing and myogenesis in…
Treadmill training using a voluntary-driven exoskeleton might be a safe approach to improve movement and exercise capacity in patients with limb-girdle muscular dystrophy, according to a small study by researchers at Ruhr University Bochum in Germany. The study, “Treadmill Training with HAL Exoskeleton — A Novel Approach for Symptomatic…
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 chemical structure of a drug can make the entire difference between a success and a failure in drug development. Of two drugs that used the same approach to treat Duchenne muscular dystrophy — Exondys 51 (eteplirsen) and Kyndrisa (drisapersen) — only one became approved. Researchers from The Ohio…
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