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…
News
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…
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…
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…
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