Using CRISPR-Cpf1 gene editing, researchers have fixed mutations that cause a form of muscular dystrophy in cultured human cardiomyocytes and a mouse model.
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Marathon Pharmaceuticals pauses rollout of DMD drug in US
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FDA Approves EMFLAZA™ (deflazacort) Tablets and Oral Suspension for the Treatment of Duchenne Muscular Dystrophy in Patients 5 Years and Older
Marathon Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has granted approval of EMFLAZA™ (deflazacort) for the treatment of Duchenne muscular dystrophy in patients 5 years and older.
Sarepta Therapeutics Enters into Research Agreement and Option Agreement with Nationwide Children’s Hospital for Microdystrophin Gene Therapy Program
The initial trial, expected to go into Phase 1/2a trial in late 2017, will be conducted at Nationwide Children’s. Parent Project Muscular Dystrophy (PPMD) has committed 2.2 million dollars to the trial, with support from additional Duchenne foundations and families. Sarepta has committed to the trial through a separate research agreement with Nationwide Children’s, and has an exclusive option to license the program. PPMD’s grant provided incentive for Sarepta to help expand and accelerate this opportunity.
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Sarepta Therapeutics Enters into License Agreement with Nationwide Children’s Hospital for Galgt2 Gene Therapy Program
The experimental program explores the potential surrogate gene therapy approach to Duchenne muscular dystrophy. As a “surrogate gene therapy approach”, the gene therapy looks to induce genes that make proteins that can perform a similar function as dystrophin, with the goal of producing a muscle cell that can function normally even when dystrophin is absent. It has the potential to be used broadly in several muscular dystrophies
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