A combination of two treatments significantly approved muscle function in mice with Duchenne muscular dystrophy (DMD), a study shows. Since both treatments are already approved, the findings “may open up new therapeutic avenues for Duchenne muscular dystrophy and possibly other neuromuscular diseases,” the researchers said.
The European Commission (EC) has decided to renew the conditional marketing authorization for Translarna (ataluren) to treat certain nonsense mutation Duchenne muscular dystrophy (nmDMD) patients. PTC Therapeutics can now market Translarna for nmDMD patients who are 5 or older, mobile, and living in the European Union, Iceland, Liechtenstein, and Norway. The decision follows a…
Long-term therapy with daily glucocorticoid therapy can improve clinical outcomes of young patients with Duchenne muscular dystrophy (DMD) without serious side effects, according to recent research. In “Long-Term Outcome of Interdisciplinary Management of Patients with Duchenne Muscular Dystrophy Receiving Daily Glucocorticoid Treatment,” researchers performed a retrospective study with 97 DMD…
The nonprofit Duchenne muscular dystrophy (DMD) research and advocacy organization Parent Project Muscular Dystrophy (PPMD) awarded a $2.2 million grant to Jerry Mendell, MD, PhD; co-principal investigator Louise Rodino-Klapac, PhD; and Nationwide Children’s Hospital in Columbus, Ohio, where they both work. The funding will support…
The study of rare diseases is typically short-changed with research funding, and the creation and sharing of tools to fight these disorders is the mission of a new initiative launched at the University of North Carolina at Chapel Hill. The UNC Catalyst for Rare Diseases project, funded…
Researchers found notable differences in the access to appropriate care for Duchenne’s muscular dystrophy (DMD) patients in seven European countries. In a survey known as CARE-NMD, patients in DMD registries in the countries — Bulgaria, the Czech Republic, Denmark, Germany, Hungary, Poland and the United Kingdom — were questioned as to care…
Children with special healthcare needs, such as those with muscular dystrophy (MD), benefit from at-home care, but at high costs to their families. In fact, uncompensated medical care in the U.S. costs theses families up to $36 billion annually, a new comprehensive national study reported. The study, published in…
A European Medicines Agency (EMA) committee is beginning a review of Sarepta Therapeutics‘ request for conditional approval of eteplirsen as a treatment for Duchenne muscular dystrophy (DMD) patients with a confirmed mutation of the dystrophin gene that can be overcome by exon 51 skipping. The Committee for Human Medicinal Products (CHMP) validated a Marketing Authorization application (MAA)…
Scientists are using the CRISPR-Cas9 gene-editing technique to excise the defective sequence of the gene underlying Duchenne muscular dystrophy (DMD) and replace it with the correct sequence for restoring healthy gene function in these patients. “We want to use genetically corrected stem cells to replace the stem cell pool and…
CureDuchenne Ventures is investing in Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS), a new specialized contract research organization. TRiNDS is hived off from the Cooperative International Neuromuscular Research Group (CINRG), an academic consortium that studies causes, pathogenesis and clinical outcomes of neuromuscular disorders, and conducts well-controlled clinical studies…
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