The European Medicines Agency (EMA) has started reviewing an application to market givinostat for Duchenne muscular dystrophy (DMD). “Now that our market authorization application is under review, we are looking forward to working with the regulatory authorities to bring this treatment option to patients,” Paolo Bettica, MD, PhD,…
News
Pamrevlumab, an antibody in FibroGen’s pipeline, did not improve motor skills in boys with Duchenne muscular dystrophy (DMD), ages 6 to 11, who could walk, according to top-line data from a Phase 3 study. The LELANTOS-2 (NCT04632940) trial tested against a placebo pamrevlumab’s safety and how…
September will be a busy month for patients, supporters, doctors, and researchers in the muscular dystrophy (MD) community as they recognize National Muscular Dystrophy Awareness Month. Raising awareness and critical funds for the more than 300,000 families across the country with MD and related neuromuscular diseases is the goal…
Blocking the activation of a channel in mitochondria, the cells’ powerhouses, may be a promising way to stop muscular dystrophy (MD) progression in a mouse model of the disease. “We have isolated the primary disease-causing component of muscular dystrophy to the mitochondrial permeability pore,” Jeffery Molkentin, PhD, co-executive director…
More than 420 “Fill the Boot” events will raise money throughout Labor Day weekend to advance research and care for muscular dystrophy. The campaign results from a partnership between the Muscular Dystrophy Association (MDA) and the International Association of Fire Fighters (IAFF). The firefighters go to…
Deleting exon 51 in the DMD gene — which mimics exon 51 skipping therapies — reversed all signs of Duchenne muscular dystrophy (DMD) in a pig model lacking exon 52, a severe disease-causing mutation in people, a study reported. Pigs missing both exon 51 and 52 had normalized dystrophin…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AOC 1044, an experimental exon 44-skipping therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Avidity Biosciences. Orphan drug designation is given to therapies with the potential to treat rare disorders, those that affect fewer…
A new project hopes to improve data collection related to Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), with the aim of accelerating research for both diseases. The effort is a collaboration between the Duchenne Data Foundation (DDF) and the Duchenne Regulatory Science Consortium (D-RSC), a…
A 5-year-old boy has received an infusion of Elevidys, the recently approved gene therapy for Duchenne muscular dystrophy (DMD), at Nationwide Children’s Hospital, where the treatment was first investigated. Gideon Griffiths of Bellefontaine, Ohio, who was diagnosed with DMD at birth, is among the first group of patients nationwide…
The first person with limb-girdle muscular dystrophy type 2I/R9 (LGMD 2I/R9) has been dosed with the gene therapy AB-1003 in a clinical trial, according to an announcement from the therapy’s developer Asklepios BioPharmaceutical (AskBio). “The first limb-girdle muscular dystrophy 2I/R9 patient dosed in the LION-CS101 clinical trial is…
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