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…
News
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…
New discoveries in the laboratory are leading to exciting, new possibilities for developing the next generation of Muscular dystrophy therapies. Recently, researchers found that by targeting muscle repair, rather than that the genetic defect that is the underlying cause for muscular dystrophy, they may be able to treat the…
Clinical stage biopharmaceutical company Akashi Therapeutics recently acquired the rights for a peptide therapy to regulate the calcium levels in the muscles, a symptom experienced by Duchenne muscular dystrophy (DMD) patients that causes loss of function and other pathologies. The treatment, called GsMTx-4, was developed by the Buffalo start-up Tonus Therapeutics, based…
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…
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