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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 (Â鶹Éç) 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 (Â鶹Éç) 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 (Â鶹Éç) 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 (Â鶹Éç) 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 (Â鶹Éç), 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 (Â鶹Éç) 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…

Patients with Duchenne muscular dystrophy (Â鶹Éç) and their caregivers now have a new tool to use to find an array of goods, programs, and services that can assist them with a variety of needs. The resource finder is part of the Duchenne Family Assistance Program (DFAP), an effort the…

For more than four decades, comedian Jerry Lewis hosted the Â鶹Éç Labor Day Telethon on behalf of the Muscular Dystrophy Association. That annual event helped the Â鶹Éç become the nation’s largest non-government source of funding for neuromuscular disease research, with more than $1.4 billion disbursed since its establishment 68…

Santhera Pharmaceuticals intends to file for a conditional marketing authorization with the European Medicines Agency (EMA) for idebenone — to be marketed as Puldysa — as a treatment for respiratory dysfunction in Duchenne muscular dystrophy (Â鶹Éç). As it continues to gather data on the therapy, the company…