Researchers from the University of Texas Southwestern Medical Center in Dallas have corrected Duchenne muscular dystrophy (DMD) mutations in human cells and experimental animal models using a new genome editing method called CRISPR-Cpf1. These new findings offer hope in permanently eliminating genetic mutations and rescue abnormalities linked to DMD. The study, “…
News
New York-based Bristol-Myers Squibb announced two separate agreements with Biogen of Cambridge, Mass., and Switzerland’s Roche to license compounds that aim to treat progressive supranuclear palsy (PSP) and Duchenne muscular dystrophy (DMD). Tau protein contributes to the stability of basic cellular structures known as microtubules to transport molecules. Abnormal addition of…
Three grassroots foundations started by the families of boys with Duchenne muscular dystrophy (DMD) have come together to award a $100,000 research grant to the Kennedy Krieger Institute’s Center for Genetic Muscle Disorders to study potential therapies. The Kennedy Krieger Institute in Baltimore provides services for children…
A small protein called Myomixer plays a crucial role in the development of skeletal muscle, the type of muscles the body uses to move, according to a study that has implications for muscular dystrophy (MD). The research at the University of Texas Southwestern Medical Center could lead to treatments for the…
CureDuchenne has announced its enthusiastic support and sponsorship of California Senate bill SB 643, which adds Duchenne muscular dystrophy to California’s Genetically Handicapped Persons Program (GHPP) list of eligible medical conditions. The bill, introduced Feb. 17 by Richard Pan (D) — a pediatrician and state senator representing the…
Parent Project Muscular Dystrophy (PPMD), a nonprofit group dedicated to ending Duchenne muscular dystrophy (DMD), has named the Neuromuscular Research Center at the University of California Davis Department of Pysical Medicine and Rehabilitation a certified Duchenne care center. PPMD’s Certified Duchenne Care Center Program,…
Eighteen-year-old Brandon Martz will travel from Michigan to Washington, D.C., to raise awareness for muscular dystrophy (MD) and see the nation’s capital in one “final Dream trip” with the support of Dream Foundation. Brandon was diagnosed with Duchenne muscular dystrophy (DMD) when he was four and was…
Catabasis Pharmaceuticals is facing challenges in its efforts to develop Edasalonexent (CAT-1004) — an oral drug that targets NF-kB, a protein activated in Duchenne muscular dystrophy (DMD) — to treat the rare degenerative disease. Edasalonexent is being evaluated in MoveDMD, an ongoing Phase 1/2 clinical trial (NCT02439216) consisting of…
A gene therapy approach that Benitec Biopharma is developing for oculopharyngeal muscular dystrophy (OPMD) brought impaired muscle strength in mice back to healthy levels and decreased their muscle fibrosis, or scarring, according to a preclinical trial study. Benitec said its approach could become an effective strategy for treating the rare genetic…
Researchers investigated swallowing dysfunctions in Becker muscular dystrophy (BMD) patients and found they have swallowing problems similar to those observed in patients with Duchenne muscular dystrophy (DMD). These observations are important not only for a better understanding of BMD progression, but also to improve the standard care of these patients. The…
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