Jan 31, 2025 · Phase 1/2 trial of exon 44 skipping therapy for DMD cleared in UK. Entrada Therapeutics announced it has been cleared in the U.K. to start a Phase 1/2 clinical trial of ENTR-601-44, at increasing doses, in Duchenne muscular dystrophy (DMD) patients with a mutation in the DMD gene amenable to exon 44 skipping.

Jan 2, 2025 · Read the story here. No. 8 – PPMD conference highlighted DMD gene therapy and treatments. As part of our coverage of the 2024 Parent Project Muscular Dystrophy (PPMD) Annual Conference, we reported on a session discussing up-and-coming treatments, including gene therapies and approaches to regenerate muscle cells. As the DMD gene that is mutated in people with Duchenne is too big to fit ...

1 day ago · The “impressive results” are a “pivotal moment” for developer Cumberland Pharmaceuticals, “and, more importantly, for the DMD community,” A.J. Kazimi, CEO of Cumberland, said in a company press release.. The Phase 2 study, FIGHT DMD (NCT03340675), enrolled 41 male patients with DMD ages 7 and older.Participants were randomly assigned to receive one of two doses of ifetroban (150 mg ...

3 days ago · The goal of exon-skipping therapy is to allow the body to make a shorter but functional form of the dystrophin protein by skipping over a specific exon on the DMD gene. ENTR-601-44 is being developed for patients who have a genetic mutation amenable to exon 44 skipping. With ENTR-601-44, exon 44 is “patched” with a short strand of DNA, called an oligonucleotide, that allows it to be ...

FDA awards fast track designation to DM1 treatment DYNE-101. The U.S. Food and Drug Administration (FDA) has granted fast track designation to DYNE-101, a treatment candidate for myotonic dystrophy type 1 (DM1) — in which disease symptoms begin during adulthood — that’s now being tested in a clinical trial involving DM1 patients.

Jan 2, 2024 · No. 10 – With EMBARK trial data, Sarepta seeks to expand Elevidys’ approval. Elevidys (delandistrogene moxeparvovec-roki) is a one-time gene therapy approved in the U.S. for certain patients with Duchenne muscular dystrophy (DMD). Its developer, Sarepta Therapeutics, plans to seek expanded approval of the treatment following top-line findings in the Phase 3 EMBARK (NCT05096221) clinical ...

Jan 28, 2025 · That’s according to top-line findings from part two of the Phase 3 EMBARK trial (NCT05096221), which evaluated Sarepta Therapeutics‘ approved gene therapy in more than 120 boys with DMD ages 4 to 7.. Significant motor function gains also were seen one year after treatment among children who began with a placebo in part one of EMBARK and a year later were treated with Elevidys, compared ...

Jan 9, 2025 · Gene-editing therapy targets DYSF, whose mutations cause LGMD type 2B/R2. MD refers to a group of disorders characterized by progressively worsening muscle weakness and wasting due to mutations in genes encoding proteins important for muscle health.. Mutations in DYSF, which encodes the production of dysferlin, are the cause of LGMD type 2B/R2.Dysferlin is important for repairing muscle fiber ...

Dec 17, 2024 · “This landmark study presents compelling biomarker data and promising signals that suggest the potential for functional stabilization with administration of sevasemten,” Craig McDonald, MD, a principal trial investigator and professor at the University of California Davis, said in a company press release.. Edgewise scientists will join Parent Project Muscular Dystrophy to …

Aug 8, 2024 · Scientific advances have helped those with DMD live longer and richer lives. A number of treatments can help with symptoms and even slow disease progression including gene therapy and exon-skipping therapies.. But not every DMD treatment is pharmaceutical. Treatment can encompass physical therapy (PT), counseling, assistive devices, home adaptations, taking part in clinical trials, and even ...