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…
Using a genetic engineering tool to restore dystrophin while raising levels of utrophin, a similar protein, leads to better improvements in muscle function than either approach alone, research in a mouse model of Duchenne muscular dystrophy (DMD) reports. The study, “The potential of utrophin and dystrophin…
Cugamycin, a small molecule compound targeting the disease-causing RNA repeats in people with myotonic dystrophy type 1 (DM1), showed promise in improving muscle defects — without evident side effects — in an early study using mouse and cell models, researchers report.
Sarepta Therapeutics‘ casimersen (SRP-4045), one of the company’s investigational exon-skipping therapies for Duchenne muscular dystrophy (DMD), showed promising results in an interim analysis of an ongoing Phase 3 clinical trial. These positive data are expected to support the submission of a new drug application (NDA) with the…
New nine-month data on four boys with Duchenne muscular dystrophy (DMD) enrolled in Study-101 testing Sarepta Therapeutics‘ micro-dystrophin gene therapy continues to show “very encouraging” results, company officials said. These updated data, given in a recent investors’ webinar, show the gene therapy resulted in 81.2% increase in dystrophin protein…
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