The agency that plays the key role in European Union therapy authorizations has recommended against approval of Raxone (idebenone) as a Duchenne muscular dystrophy (DMD) treatment. Santhera said in a press release that it plans to appeal the Committee for Medicinal Products for Human Use’s decision, which covers Raxone in DMD patients not being treated…
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In a recent webinar, Dr. Jerry Mendell with Nationwide Children’s Hospital rolled out plans for a Phase 1/2a clinical trial of a new gene therapy for Duchenne’s muscular dystrophy (DMD). Final go-ahead for the trial awaits U.S. Food and Drug Administration (FDA) approval of an Investigational New Drug (IND) application made for…
Young muscle stem cells in patients with Duchenne muscular dystrophy have shortened telomeres, causing these cells to be less able to build new muscle, according to new research. A full discussion of the results of this study, “Single Stem Cell Imaging and Analysis Reveals Telomere Length Differences in Diseased Human and Mouse…
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…
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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