The U.S. Food and Drug Administration (FDA) is expected to decide by Feb. 28, 2027, whether to grant traditional approval to Amondys 45 (casimersen) and Vyondys 53 (golodirsen), two exon-skipping therapies designed to treat certain people with Duchenne muscular dystrophy (DMD). Both medications are currently approved in…
News
Amid its ongoing review of the cell therapy deramiocel for people with Duchenne muscular dystrophy (DMD), the U.S. Food and Drug Administration (FDA) has scheduled a meeting with Capricor Therapeutics, its developer, to discuss the company’s resubmission of an application…
The Department of Justice (DOJ) has released a memorandum arguing that states are not required to provide in-home or community-based care to people with disabilities, including those with muscular dystrophy (MD). The move drew sharp condemnation from the Muscular Dystrophy Association (MDA), which stated in a press…
The CITGO Lake Charles Refinery raised more than $750,000 to support the Muscular Dystrophy Association (MDA) at its 41st annual MDA Golf Classic, an annual golf fundraiser. Since 1985, CITGO Lake Charles has raised $8.5 million for the MDA. The total amount raised this year — $756,800 — set…
Health Canada has accepted and granted priority review to Italfarmaco’s application seeking the approval of oral givinostat, sold in the U.S. under the brand name Duvyzat, to treat Duchenne muscular dystrophy (DMD). The priority review status is reserved for therapies that could significantly improve the benefit-risk profile over…
The experimental medication delpacibart braxlosiran (del-brax) reduced biological markers of facioscapulohumeral muscular dystrophy (FSHD) disease activity in a Phase 1/2 clinical trial, meeting the study’s goals, according to developer Novartis. Based on results from earlier parts of the trial, FORTITUDE (NCT05747924), investigators selected a dosage for…
The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to GEn1E Lifesciences‘ GEn-1123 as a potential treatment for Duchenne muscular dystrophy (DMD). The designations aim to incentivize the development of treatments for rare disorders, which are those affecting fewer than 200,000 people…
Among women who carry mutations that can cause Becker muscular dystrophy (BMD) or Duchenne muscular dystrophy (DMD), measures of heart health — important given that heart muscle damage is a key driver of death in most people with these…
Dyne Therapeutics expects to report top-line data in early 2027 from its Phase 1/2 ACHIEVE trial testing zeleciment basivarsen (z-basivarsen), an experimental treatment for myotonic dystrophy type 1 (DM1). The company announced that enrollment is now complete for this key group of patients. If the results are positive,…
Adults with muscular dystrophy (MD) in the U.S. report widespread gaps in sexual and reproductive healthcare, according to surveys and interviews. Such gaps included a lack of private clinical time, provider discomfort, and unaddressed concerns about fertility and relationships. At the same time, participants indicated a strong desire for…
Recent Posts
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- I now appreciate it when someone says, ‘You have your hands full’
- FDA review begins for full approval bids on 2 Duchenne therapies
- The search for better accommodations for my disease progression continues
- July meeting date set for FDA, developer to talk deramiocel for DMD