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…
A new study presented at the EuroSpine Annual Meeting on October 1 – 3 in Lyon, France reported the successful placement of a single rod with hybrid fixation to correct scoliosis in children with Duchenne’s muscular dystrophy. Duchenne muscular dystrophy (DMD) is a genetic disease caused by mutations in…
A recent study entitled “Proteasome inhibitors increase missense mutated dysferlin in patients with muscular dystrophy” published in Science Translational Medicine reports increased expression of the protein dysferlin, via inhibiting proteasome activity, may become a new therapeutic treatment for patients with muscular dystrophy. Muscular dystrophies (as…
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