News

The U.S. Food and Drug Administration (FDA) accepted NS Pharma‘s new drug application for viltolarsen, a potential treatment for Duchenne muscular dystrophy (DMD) patients who are amenable to exon 53 skipping, the company and its parent, Nippon Shinyaku, said. The companies expect a decision on approval between…

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…

A new project called BIND — Brain Involvement  iN  Dystrophinopathies — seeks to better characterize how the brain is affected in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), and to find potential ways to treat neurological complications in people with those disorders. The European Union awarded…

Eighty delegates from 37 countries recently met in Athens for the 2019 Duchenne Patient Academy — a three-day series of master classes aimed at deepening global understanding of Duchenne muscular dystrophy (DMD) and coordinating advocacy strategy. The Dec. 6-8 conference, an annual event since its 2017 start, covered subjects…

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…