Patricia Inácio, PhD, science writer —

A new study entitled “Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9” reports an iPSC-based gene therapy that can correct the genetic defect underlying Duchenne Muscular Dystrophy. The study was published in the journal…

Sarepta Therapeutics, Inc. recently announced a new study to confirm the dosages of their lead drug eteplirsen as a new therapeutic for treating Duchenne muscular dystrophy-affected patients with a baseline 6-minute walk test score. Duchenne muscular dystrophy (DMD) is a form of muscular dystrophy that is caused by a mutation of the Dystrophin gene,…

A team of researchers recently presented their latest results showing how injecting cardiac stem cells in mouse models of Duchenne muscular dystrophy can reverse damage to the heart. These findings carry a new therapeutic promise to patients with muscular waisting disorders. The study was presented in the American Heart…

A study entitled “Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients” describes the positive results of using a recombinant adeno-associated virus vector to restore dystrophin levels in dogs affected with Duchenne muscular dystrophy.

A new study entitled “Identifying diagnostic DNA methylation profiles for facioscapulohumeral muscular dystrophy in blood and saliva using bisulfite sequencing” published in the Clinical Epigenetics journal designed an assay that identifies the epigenetic signature for both types of Facioscapulohumeral muscular dystrophy from a multiple collections of…

The results from Milo Biotechnology’s Follistatin Gene Therapy were published in the Molecular Therapy journal showing therapy efficacy in increasing muscle strength and function in Becker’s muscular dystrophy patients. The study is entitled “A Phase I/IIa Follistatin Gene Therapy Trial for Becker Muscular Dystrophy.” The study followed…

PTC Therapeutics, Inc. announced the results of a Phase 2b clinical trial entitled “Ataluren treatment of patients with nonsense mutation dystrophinopathy” in the October issue of Muscle & Nerve, demonstrating that Translarna™ (ataluren) improved clinical outcomes of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) in males five…

A new study entitled “NBD delivery improves the disease phenotype of the golden retriever model of Duchenne muscular dystrophy” published in Skeletal Muscle journal reports the results of a trial with an inhibitor of NF-κB, (NEMO)-binding domain (NBD) peptide, in a canine model…

A new study entitled “A Phase I/IIa Follistatin Gene Therapy Trial for Becker Muscular Dystrophy” published in the Molecular Therapy journal, describes the successful results with Follistatin as a therapy for treating dystrophin-deficient muscle diseases. Becker muscular dystrophy (BMD) is a type of dystrophin-deficient…

A study entitled “Left ventricular systolic function and the pattern of late-gadolinium-enhancement independently and additively predicts adverse cardiac events in muscular dystrophy patients” published in the September issue of the Journal of Cardiovascular Magnetic Resonance report two new independent predictors for adverse cardiac events in Duchenne and…