Patricia Inácio, PhD, science writer —

This year, the Muscular Dystrophy Association (MDA) will celebrate MDA National Muscular Dystrophy Awareness Month, held every September, with a series of online events. The events will be held virtually for the first time to ensure maximum participation, while promoting social distancing and other safety measures for people with…

The U.S. Food and Drug Administration (FDA) is maintaining the clinical hold on the Phase 1/2 trial testing Solid Biosciences’ gene therapy SGT-001 for people with Duchenne muscular dystrophy (DMD), the company announced. In a letter to Solid, the FDA has requested more information about the gene therapy’s…

One-year treatment with CAP-1002, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy (DMD) significantly improves respiratory, cardiac and upper limb function in boys and young men at advanced stages of the disease, top-line data from the HOPE-2 clinical trial show. “I am incredibly pleased with the outcome of the…

Treatment with Vyondys 53 (golodirsen) for 48 weeks increases levels of dystrophin protein by 16-fold in boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, updated results of a Phase 1/2 trial show. Also, the newly produced protein was correctly located in the membrane of muscle…

Treatment for six months with Increlex (mecasermin), an injectable form of insulin-like growth factor-I (IGF-I), enhanced growth in boys with Duchenne muscular dystrophy (DMD), a Phase 1/2 trial shows. However, the hormone therapy had no effects on muscle function. The study, “Recombinant human insulin‐like growth factor‐1…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

A small molecule, trichostatin A (TSA), increased the levels of a dystrophin-related protein known as utrophin, and improved the structure and function of muscles in a mouse model of Duchenne muscular dystrophy (DMD), a study found. The study, “High-throughput identification of post-transcriptional utrophin up-regulators for Duchenne muscle…

GPM Investments, the sixth largest convenience store chain in the U.S., is running its “Tis the Season for Giving” holiday campaign throughout December to support the Muscular Dystrophy Association (MDA). Since 2012, the company’s partnership with the association has generated more than $1.2 million, which has been used…

Loss of MICU1, a key regulator of calcium balance in cells, was seen to affect muscle function and repair in a study in mice and cells from people lacking this mitochondrial protein. These findings support the involvement of MICU1 in neuromuscular disorders, its researchers said, and its potential as a…

Treatment with a cell therapy candidate called dystrophin expressing chimeras (DECs) increased dystrophin levels in heart muscle and improved cardiac function in mice with Duchenne muscular dystrophy (DMD), a study has found. This cell therapy, by Dystrogen Therapeutics, will be tested in a clinical trial involving DMD patients,…