Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…
News
NS Pharma launched an expanded access program (EAP) in the U.S. to allow certain Duchenne muscular dystrophy (DMD) patients to gain access to its investigational exon-skipping therapy viltolarsen. The program will allow physicians to prescribe viltolarsen to U.S. DMD patients amenable to exon 53 skipping who meet other eligibility criteria, at…
Machine learning could be used to help diagnose specific forms of muscular dystrophy (MD) using magnetic resonance imaging (MRI) scans, a study suggests. The study, “Accuracy of a machine learning muscle MRI-based tool for the diagnosis of muscular dystrophies,” was published in the journal…
Capricor Therapeutics is expanding the development of exosome-based technologies that are being tested as a potential way to treat Duchenne muscular dystrophy (DMD). Capricor’s lead candidate for DMD is CAP-1002, which is composed of cardiosphere-derived cells (CDCs). These are cardiac progenitor cells, meaning they are able to…
Oral treatment with AdipoRon reduced inflammation in muscle, promoted muscle fiber proliferation, and improved muscle and motor function in a mouse model of Duchenne muscular dystrophy (DMD), a study showed. Anti-inflammatory effects were also seen in muscle cells from people with DMD. The scientists said AdipoRon has the potential…
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…
The Muscular Dystrophy Association (MDA) is launching MDA Let’s Play, a platform to raise funding and awareness about muscular dystrophy and related neuromuscular disorders through gaming. “We’ve always been an iconic American charity with innovative fundraising programs to achieve our mission of transforming lives — from the telethon to…
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 sixth annual Napa in Newport wine auction raised more than $1 million to support CureDuchenne’s mission to extend and enhance the lives of people with Duchenne muscular dystrophy (DMD) and find a cure for the disorder, the nonprofit group said. Held Feb. 29 at the…
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…
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