News

Orsini Pharmaceutical Services is now providing Sarepta Therapeutics’ newly approved therapy Vyondys 53 (golodirsen) to treat people with Duchenne muscular dystrophy (DMD) responsive to exon 53 skipping…

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

Expecting to award roughly $18 million in grants in 2020 to support research across neuromuscular diseases, the Muscular Dystrophy Association (MDA) continues to be a leader in efforts to better treat and otherwise ease life for those touched by muscular dystrophy and numerous other, often rare, illnesses.

Gene therapies — after a tragedy kicked them back to the lab some two decades ago — are beginning to come into their own, making possible work in gene-targeted and combination treatments for neuromuscular diseases once unimaginable. But many challenges — from the likelihood of no “one and done” gene…

Blocking the protein receptor IGF2R could promote muscle regeneration and improve muscle strength in people with Duchenne muscular dystrophy (DMD), a study in mice suggests. The study, “Blockade of IGF2R improves muscle regeneration and ameliorates Duchenne muscular dystrophy,” was published in the journal EMBO…

The gene-editing tool CRISPR/Cas9 was used to correct defects in the DMD  gene and restore dystrophin protein production,  lengthening the lives of pigs in a model of Duchenne muscular dystrophy (DMD) and altering heart cells from a patient to make them less prone to irregular beats, researchers report. The scientists…

Longtime Muscular Dystrophy Association (MDA) supporter CN Brown Company, a CITGO Petroleum Corporation (CITGO) marketer, raised more than $300,000 for the MDA’s Maine chapter in 2019. “Our family’s commitment to helping win the fight for families affected by MDA has remained strong for generations,” Jeff Jones, CN…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to losmapimod, an investigational oral therapy for people with facioscapulohumeral muscular dystrophy (FSHD). This designation offers Fulcrum Therapeutics financial support to develop this treatment, as well as a waiver from the FDA’s fees and…

Kevin Schaefer hadn’t been in an airport since he was 4 years old, so he had been looking forward to flying from his home in Cary, North Carolina, to Anaheim, California, in June for the 2019 Cure SMA Conference. As it turned out, his experience didn’t go as expected.

Altering connexin 43 (Cx43) — a protein involved in heart function — in cardiac muscle cells protected against heart disease in a mouse model of Duchenne muscular dystrophy (DMD), a study found. These results suggest targeting Cx43 may prevent heart disease in people with DMD. The study, “…