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…
A new clinical trial is seeking about 80 people with limb-girdle muscular dystrophy type 2i (LGMD2i) to gather information on this disease’s progression that may help in advancing potential therapies and in establishing a biomarker. The observational study (NCT04202627) is now enrolling patients, ages 10 to 65, at…
PerkinElmer Genomics has launched a new test that is able to accurately detect the shortening of the D4Z4 gene region which causes facioscapulohumeral muscular dystrophy (FSHD) type 1. FSHD affects approximately one in 10,000 to one in…
Supplementing the diet with the amino acid L-arginine may improve gut function in people with Duchene Muscular Dystrophy (DMD), a study in mice suggests. The study, “Spatiotemporal Mapping Reveals Regional Gastrointestinal Dysfunction in Mdx Dystrophic Mice Ameliorated by Oral L-arginine Supplementation,” was published in the …
Addressing changes in cellular metabolism and oxidative damage may lead to muscle regeneration in Duchenne muscular dystrophy and Becker muscular dystrophy, a study has found. The study, “Comparative proteomic analyses of Duchenne muscular dystrophy and Becker muscular dystrophy muscles: changes contributing to preserve muscle…
In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…
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