No treatments are currently approved and available for limb-girdle muscular dystrophy type 2i (LGMD2i) in the U.S., but that may change later this year. The U.S. Food and Drug Administration (FDA) has accepted an application from Bridgebio Pharma seeking approval for its experimental oral therapy BBP-418. The…
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Seeking to build on the positive results seen among boys with Duchenne muscular dystrophy (DMD) treated with its experimental exon-skipping therapy zeleciment rostudirsen — z-rostudirsen, formerly known as DYNE-251 — in an early clinical trial, Dyne Therapeutics has launched a global confirmatory study, dubbed FORZETTO. This new Phase 3…
An experimental gene therapy called ATA-200 has shown promising safety and efficacy results in an early clinical trial for children with limb-girdle muscular dystrophy type R5 (LGMDR5). Long-term data from the first two treated patients suggest the therapy is working as expected, with no serious side effects, according to…
The Phase 3 portion of a clinical trial testing RGX-202, an investigational gene therapy for Duchenne muscular dystrophy (DMD), met its main goal, the therapy’s developer, Regenxbio, announced in a press release. Steve Pakola, MD, Regenxbio’s chief medical officer, said the data “support the potential of RGX-202…
Bridgebio is providing $100,000 to the Muscular Dystrophy Association (MDA) to advance projects aimed at improving multidisciplinary care for people with limb-girdle muscular dystrophy (LGMD). The awards will go to initiatives at Stanford Health Care and the University of Minnesota that aim to make LGMD care more…
Duchenne Parent Project Spain is investing €247,000 (just over $290,000 USD) in a project that aims to create a new laboratory model to study how the heart is affected in people with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). The “heart-on-a-chip” platform will allow researchers…
Epicrispr Biotechnologies is partnering with Forge Biologics to support the development of EPI-321, Epicrispr’s one-time epigenetic treatment candidate for facioscapulohumeral muscular dystrophy (FSHD). The treatment uses epigenetics — chemical modifications that can alter gene activity without changing a gene’s DNA sequence — to turn off the abnormally-activated…
Regulators in the European Union have granted orphan drug designation to SGT-003, a gene therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Solid Biosciences. The treatment candidate, already awarded orphan drug status in the U.S., aims to preserve muscle health in boys with DMD. This…
CureDuchenne will host its annual Futures National Conference, a four-day event focused on bringing education, connection, and hope to the Duchenne and Becker muscular dystrophy (MD) community. The event will be held on May 21-24 at the JW Marriott Orlando, Grande Lakes. It will highlight the latest…
The Committee for Medicinal Products for Human Use (CHMP) has recommended that approval of Agamree (vamorolone) in the European Union be expanded to include people with Duchenne muscular dystrophy (DMD) as young as 2. The CHMP is an arm of the European Medicines Agency (EMA) tasked with reviewing…
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