News

Catabasis Pharmaceuticals and the Jain Foundation have started a preclinical research collaboration to study edasalonexent as an oral treatment candidate for dysferlinopathy, a group of muscle diseases that includes limb-girdle muscular dystrophy (LGMD) type 2B. Edasalonexent, formerly CAT-1004, is a small molecule designed…

Dyne Therapeutics announced that it has joined in supporting an ongoing global and observational natural history study to better inform the development of treatments for facioscapulohumeral muscular dystrophy (FSHD). The company’s support — the amount was not disclosed — will help to bring new sites across Europe into…

New nine-month data from a Phase 1/2 trial show that a single administration of the gene therapy SRP-9003 (formerly, MYO-101) at low dose significantly improved functional measures and lowered the levels of a biomarker of muscle damage in three children with limb girdle muscular dystrophy (LGMD) type 2E. The trial (…

Nippon Shinyaku filed a request for the approval of viltolarsen (NS-065/NCNP-01) to treat Duchenne muscular dystrophy amenable to exon 53 skipping with the U.S. Food and Drug Administration (FDA), the company announced in a press release. The request came in the form of a rolling new drug application (NDA) whose…

Parent Project Muscular Dystrophy (PPMD) announced the screening of a first baby for Duchenne muscular dystrophy (DMD) within the organization’s pilot newborn screening program in New York. The $5 million pilot program, launched in collaboration with the American College of Medical Genetics and Genomics (ACMG) and New York State,…

Cumberland Pharmaceuticals announced that it has been awarded a $1 million grant by the U.S. Food and Drug Administration (FDA) to support a Phase 2 clinical trial into oral ifetroban as potential treatment of heart disease associated with Duchenne muscular dystrophy (DMD). The award marks the first time…

About 700 people have registered to attend CureDuchenne’s Futures conference taking place Oct. 11-13 in suburban Los Angeles — more than six times the number of participants at the November 2018 Futures meeting near Boston. The annual gathering, to be held at Anaheim’s Disneyland Hotel, will be the largest…

Expanding certification of clinics that provide optimal Duchenne muscular dystrophy care, Parent Project Muscular Dystrophy (PPMD) has announced its first Adult Certified Duchenne Care Center. An extension of the nonprofit’s five-year-old, 26-member Certified Duchenne Care Center Program (CDCCP), the neuromuscular clinic at University of Missouri Health…

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…

Three new animal-model tests of a potential gene therapy for oculopharyngeal muscular dystrophy (OPMD), known as BB-301, are planned, Benitec Biopharma announced. Results are expected to support a request for a Phase 1 clinical trial in patients. OPMD results from a faulty PABPN1 gene, leading to a protein…