The nonprofit group CureDuchenne won $50,000 at a recent Shark Tank-like competition in Irvine, California, to pitch scientific projects. The “RARE Battle of the Brains” competition, held Sept. 14, was organized by Scientist.com, a marketplace for scientific services, and Global Genes, a rare disease patient advocacy group. CureDuchenne won the…
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Synpromics and Solid Biosciences have established a collaborative agreement to develop new potential gene therapies for the treatment of Duchenne muscular dystrophy (DMD). According to the partnership terms, Solid Biosciences will gain access to muscle-selective promoter candidates developed by Synpromics, which are…
PTC Therapeutics shared additional data from a Phase 3 trial of Translarna (ataluren), showing that the treatment slightly slowed disease progression in ambulatory boys with Duchenne muscular dystrophy. The data was presented at the recent American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting in Phoenix, Arizona.
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Phrixus, DMD Groups Plan First Clinical Trial of Potential Duchenne Therapy Already Used in Europe
Phrixus Pharmaceuticals and several Duchenne muscular dystrophy (DMD) organizations will jointly conduct the first open-label trial of Poloxamer-188 NF, the active ingredient in Carmeseal-MD, to treat DMD in patients who can no longer walk. The trial, to take place at Cincinnati Children’s Hospital, will evaluate the drug’s performance on respiratory…
For the ninth year in a row, “Coach to Cure MD” and Parent Project Muscular Dystrophy (PPMD) will mark Duchenne Awareness Month by using college football games to raise awareness of Duchenne muscular dystrophy (DMD) and money to promote research into treatments and a possible cure. The effort, a partnership between the…
The agency that plays the key role in European Union therapy authorizations has recommended against approval of Raxone (idebenone) as a Duchenne muscular dystrophy (DMD) treatment. Santhera said in a press release that it plans to appeal the Committee for Medicinal Products for Human Use’s decision, which covers Raxone in DMD patients not being treated…
In a recent webinar, Dr. Jerry Mendell with Nationwide Children’s Hospital rolled out plans for a Phase 1/2a clinical trial of a new gene therapy for Duchenne’s muscular dystrophy (DMD). Final go-ahead for the trial awaits U.S. Food and Drug Administration (FDA) approval of an Investigational New Drug (IND) application made for…
Young muscle stem cells in patients with Duchenne muscular dystrophy have shortened telomeres, causing these cells to be less able to build new muscle, according to new research. A full discussion of the results of this study, “Single Stem Cell Imaging and Analysis Reveals Telomere Length Differences in Diseased Human and Mouse…
A University of Utah medical professor has received a $600,000 Muscular Dystrophy Association grant to conduct a clinical trial aimed at understanding how congenital myotonic dystrophy develops over time. The three-year grant will allow Dr. Nicholas Johnson, an assistant professor of neurology, pediatrics, and pathology, to study what scientists call the natural history…
The first patients with Duchenne muscular dystrophy (DMD) have been enrolled into the U.K.’s Early Access to Medicines Scheme (EAMS) for Santhera Pharmaceuticals’ Raxone (idebenone), giving patients access to the treatment before its regulatory approval. Raxone was included in EAMS after the Medicines and Healthcare Products Regulatory Agency (MHRA)…
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