BBP-418, an oral treatment candidate for patients with limb-girdle muscular dystrophy type 2I (LGMD2I), is being developed by ML Bio Solutions, a new subsidiary of BridgeBio Pharma, the company has announced. BridgeBio intends to start a natural history study by…
As part of an effort to heighten awareness of its work in research, treatment and care — as well as to raise funds — the Muscular Dystrophy Association (MDA) has proclaimed September MDA National Muscular Dystrophy Awareness Month. The launch dovetails with World Duchenne Awareness Day, observed annually…
Online forums have markedly altered how patients, caregivers, and healthcare professionals communicate and connect. Even when miles apart, individuals can establish supportive relationships, share experiences and information, and sometimes simply vent. Bionews Services, a leading online health, science and research publication company, has been rolling out its…
Vamorolone (VBP15), an experimental treatment for Duchenne muscular dystrophy (DMD) developed by ReveraGen Biopharma, improves muscle function of DMD patients in a dose-dependent manner, data from a Phase 2a extension study shows. The Phase 2b VISION-DMD (VBP15-004) trial (NCT03439670) for vamorolone is currently recruiting participants in…
Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…
Many people are inspired by a son or brother with Duchenne muscular dystrophy (DMD). When it comes to Danielle Edwards, she’s literally made the disease her career. Edwards, 26, is the community engagement manager at the Jett Foundation, a Massachusetts charity that helps families deal with the emotional and…
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…
Most parents of children with Duchenne muscular dystrophy and adults with this disease — who took part in an interview study arranged and owned by Parent Project Muscular Dystrophy (PPMD) — expressed “cautious optimism” about gene therapy as a potential DMD treatment, weighing potential benefits against risks and disease…
The nonprofit CureDuchenne has chosen veteran advocacy leader Erin Frey to become its first director of Duchenne muscular dystrophy (DMD) advancement efforts. In her official capacity as senior director of advocacy, Frey is charged with establishing relationships that promote CureDuchenne’s outreach, and connect Duchenne families and other…
The U.S Food and Drug Administration (FDA) has rejected Sarepta Therapeutic’s application seeking accelerated approval of golodirsen (SRP-4053) for Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping. Golodirsen (SRP-4053) was placed under priority review by the FDA in February 2019. The FDA’s complete response…
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