A new viral vector that is better than conventional vectors — tools designed to deliver genetic material into cells — at targeting muscle cells may prove useful in developing a more effective gene therapy for muscular dystrophy (MD), according to researchers. In fact, this new vector “is more than…
Benitec Biopharma is taking steps to get the needed regulatory clearances to start clinical testing in Europe and the U.S. of BB-301, its experimental gene therapy for oculopharyngeal muscular dystrophy or OPMD, a type of the genetic disease that typically first affects the eyelids and the throat. The…
Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…
A team of researchers received a $3-million grant from the Chan Zuckerberg Initiative to create a comprehensive blueprint of healthy skeletal muscle cells — whose dysfunction is associated with several rare muscle diseases, such as muscle dystrophy — as children grow. Muscular dystrophy comprises a group of more…
A myriad of activities are afoot to mark National Muscular Dystrophy Awareness Month, observed each September. The events aim to raise funds for, and heighten awareness of, muscular dystrophy (MD) and related neuromuscular disorders thought to affect more than 300,000 families across the U.S. Awareness days are set for…
The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…
The European Commission (EC) has granted orphan designation to AOC 1001, Avidity Biosciences’ investigational therapy for myotonic dystrophy type 1 (DM1). This designation is given to investigational therapies that have the potential to treat rare, life-threatening, or very serious diseases that affect less than five in 10,000 patients…
Cumberland Pharmaceuticals is recruiting boys and men for a Phase 2 study evaluating the safety, efficacy, and duration of effect of its oral ifetroban therapy for cardiomyopathy — a disease of the heart muscle — linked to Duchenne muscular dystrophy (DMD). The study, called FIGHT DMD (NCT03340675),…
The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…
MRI scans of muscle can be used as a biomarker to assess disease progression in Duchenne muscular dystrophy (DMD) and to support the evaluation of different therapies in clinical trials, an analysis showed. “The results of this study provide further support for the use of imaging as an objective,…
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