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…
A novel RNA-based drug from specialized biotech company, Sarepta Therapeutics, Inc., is experiencing a delay in its New Drug Application (NDA) from the US Food and Drug Administration’s (FDA), due to the FDA’s request for specific, additional data requirements from the company. The drug, eteplirsen, is one of the company’s leading pipeline…
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…
Insights into recent advances for Muscular dystrophy disease treatment, entitled “HDAC-regulated myomiRs control BAF60 variant exchange and direct the functional phenotype of fibro-adipogenic progenitors in dystrophic muscles” were recently published in the Genes & Development journal by Dr. Valentina Saccone. Dr. Saccone is part of Dr. Pier Lorenzo Puri’s…
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…
The Muscular Dystrophy Association (MDA) selected Merkle Response Management Group (Merkle RMG), a branch of Merkle, the nation’s largest private agency, to manage the MDA’s direct mail donation processing requirements. The MDA is the world’s leading nonprofit health agency working…
A new study published October 13 in The Journal of Cell Biology reveals that like a marching band falling out of step, muscle cells in Duchenne muscular dystrophy patients fail to perform in unison, and this breakdown of muscular synchronicity leads to proliferation of stiff fibrotic tissue…
An international research team led by University of California at San Francisco scientists has discovered that regulatory T cells (Tregs), a specialized subset of immune cells, suppress inflammation and muscle injury in a mouse model of Duchenne muscular dystrophy (DMD). The scientists say Tregs have potential as therapeutic agents…
The American Academy of Neurology (AAN) and the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) recently released new guidelines on the evaluation of MD patients by physicians, recommending that professionals should review all facts regarding symptoms, family history, ethnicity, physical exams, and lab tests in order to determine the…
Get regular updates to your inbox.