News

Next-gen gene therapy SGT-003 named FDA orphan drug

SGT-003, a next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD) being developed by Solid Biosciences, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The FDA gives this designation to therapies that are designed to improve medical care for rare disorders, specifically defined…

Agamree approved in UK for DMD patients, 4 and older

Agamree (vamorolone) has been approved in the U.K. for treating Duchenne muscular dystrophy (DMD) in patients 4 and older. This approval by the Medicines and Healthcare products Regulatory Agency (MHRA) follows a similar decision in the European Union. In the U.S., Agamree is approved for patients…

Awards presented for Race to End Duchenne

Race to End Duchenne, the signature fundraising program from Parent Project Muscular Dystrophy (PPMD), has presented Team Michael James with the 2024 “Racing for Every Future Team” award, while Michael Napoli was this year’s “Make Every Day Count” award recipient. Race to End Duchenne supports PPMD’s mission to cure…

Top 10 muscular dystrophy stories of 2023

Throughout 2023, Muscular Dystrophy News Today worked diligently to cover new scientific research, treatment developments, and clinical studies for muscular dystrophy (MD). Here are the 10 most-read stories of that year. No. 10 – With EMBARK trial data, Sarepta seeks to expand Elevidys’ approval Elevidys (delandistrogene moxeparvovec-roki) is a…

HuidaGene’s gene-editing therapy named rare pediatric drug by FDA

HuidaGene Therapeutics’ investigational gene-editing therapy, called HG302, for Duchenne muscular dystrophy (DMD) has been granted a rare pediatric drug designation by the U.S. Food and Drug Administration (FDA). This status is intended to incentivize companies to develop treatments for rare and serious or life-threatening diseases affecting people under…

Agamree approved in Europe to treat DMD boys ages 4 and older

The European Commission has approved Agamree (vamorolone), a dissociative corticosteroid — a treatment expected to have fewer side effects than standard steroid medications — for patients ages 4 and older with Duchenne muscular dystrophy (DMD). The decision applies to all member states of the European Union, plus Iceland,…