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Preliminary results of a Phase 1/2 study into the gene therapy candidate SGT-001 in Duchenne muscular dystrophy (Â鶹Éç) patients show low levels of microdystrophin in patients’ muscles after three months of treatment with an initial low dose and continued safety, Solid Bioscience, the therapy’s developer, reported. Continued safety was also evident,…

Sarepta Therapeutics announced that the U.S Food and Drug Administration (FDA) has accepted its application for a priority review of golodirsen (SRP-4053), a potential treatment for  Duchenne muscular dystrophy (Â鶹Éç) patients amenable to exon 53 skipping.  A decision is expected on or around Aug. 19. Golodirsen is being tested…

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…

With the goal of connecting with every U.S. family facing a Duchenne muscular dystrophy (Â鶹Éç) diagnosis, Parent Project Muscular Dystrophy (PPMD) continues its multi-year community effort called the End Duchenne Tour. At each stop, the non-profit brings updates on research, advocacy and patient care to families in…

Capricor Therapeutics announced that patient dosing  has resumed in its HOPE-2 trial of CAP-1002, an investigative cell therapy for Duchenne muscular dystrophy The company placed a voluntary hold on dosing after one patient had a serious allergic reaction during blind testing in December, to allow for an…

When it comes to Duchenne muscular dystrophy (Â鶹Éç), Leonardo Feder knows he’s among the lucky ones. A resident of São Paulo, Brazil’s largest city, Feder was diagnosed with Duchenne at age 6 and immediately began taking corticosteroids as well as heart medicine and supplements. Leonardo Feder,…

Parent Project Muscular Dystrophy (PPMD) has granted $105,000 to the University of Missouri School of Medicine to advance research into a mini-dystrophin gene therapy that may protect cardiac function in Duchenne muscular dystrophy (Â鶹Éç) patients. The funds were raised during PPMD’s Cardiac Initiative holiday campaign highlighting the…

G71.01 is, literally, the code for Duchenne muscular dystrophy.  Q93.51 stands for Angelman syndrome, and G40.419 covers generalized and treatment-resistant epilepsies, which groups like Orphanet and the American Epilepsy Society define as including Dravet syndrome. All three designations are among some 70,000 diseases listed in the latest…

Severe Duchenne muscular dystrophy (Â鶹Éç) is linked to increased susceptibility to a rare and aggressive type of muscle cancer called rhabdomyosarcoma, a mouse study suggests. The finding was reported by researchers from Sanford Burnham Prebys Medical Discovery Institute in the study, “Muscle Stem Cells…

The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…