A protein called epidermal growth factor (EGF) can help preserve muscle strength and increase muscle regeneration in a mouse model of Duchenne muscular dystrophy (DMD), a finding that may pave the way for new treatment strategies for DMD, researchers said. The study, “EGFR-Aurka Signaling Rescues Polarity and…
Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for healthy passengers. Imagine how much harder it is for patients with rare diseases who must get to doctors’ appointments or clinical trials that are hundreds of miles away from home.
Merosin-deficient muscular dystrophy, a type of congenital MD, might be diagnosed before birth using immunohistochemistry, an easy and quick lab test that looks for specific markers in cells from a mother’s placenta, according to a small study of pregnant women whose children are at risk for this disease. A misdiagnosis, however,…
A signaling pathway determines whether muscle stem cells differentiate and mature into working muscle cells, an early study in mice found. Targeting this pathway and its two proteins — Fam3a and Stat3, which works to stimulate Fam3a — may treat diseases or illnesses of muscles, including muscular dystrophies. Getting these stem cells to…
Fulcrum Therapeutics announced it has acquired global rights to develop and potentially market losmapimod, an investigational treatment for facioscapulohumeral muscular dystrophy (FSHD), and that it plans to open a Phase 2 clinical trial testing the compound’s safety and efficacy this year. The compound was initially developed by GlaxoSmithKline (GSK) for…
Promising safety and tolerability data from a Phase 1 trial on suvodirsen (WVE-210201), Wave Life Sciences‘ investigational exon-skipping therapy for boys with Duchenne muscular dystrophy (DMD), opens the door for the launch of new Phase 2/3 clinical trials later this year. Final results from the trial (NCT03508947) were…
While federal funding for fiscal year 2020 has not been finalized, 79 members of Congress have vowed to back Duchenne muscular dystrophy (DMD) research, patient support efforts and drug development. On its website, Parent Project Muscular Dystrophy (PPMD) heralded results of its annual bipartisan campaign, expressing optimism…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to pamrevlumab (FG-3019), FibroGen‘s potential anti-fibrosis treatment for Duchenne muscular dystrophy (DMD) patients. Pamrevlumab is a human monoclonal antibody that blocks the activity of the connective tissue growth factor (CTGF), a protein involved in tissue scarring (fibrosis) linked…
Audentes Therapeutics is expanding its pipeline of potential molecular therapies, expecting to address 80% of patients with Duchenne muscular dystrophy (DMD) and all with myotonic dystrophy type 1 (DM1). The treatment strategy, called vectorized exon skipping, uses a modified adeno-associated virus (AAV) to deliver…
A single dose of gene therapy based on CRISPR-Cas9 may successfully treat Duchenne muscular dystrophy (DMD) in the long term or even permanently, a preclinical study in mice shows. The findings shed light on safety factors that may affect the long-term efficacy of this gene therapy, and provide new evidence…
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