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网页5 天之前 · DMD treatment WVE-N531 boosts dystrophin: Interim trial data. Treatment with exon 53-skipping therapy WVE-N531 led to significant increases in muscle dystrophin levels along with signs of improved muscle health and regeneration in boys with Duchenne muscular dystrophy (DMD).
MDA 2024: Gene therapy’s benefits most pronounced in young boys
网页2024年3月7日 · That’s according to updated analyses from a Phase 1b trial (NCT03362502), in which the benefits of Pfizer‘s gene therapy candidate were most pronounced in children ages 6-7, with results more variable in those ages 8-12.. Perry Shieh, MD, PhD, of the University of California, Los Angeles, presented the findings at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific ...
DMD treatment WVE-N531 boosts dystrophin: Interim trial data
网页4 天之前 · In particular, WVE-N531 is designed to skip exon 53 of DMD, an approach estimated to benefit about 8%-10% of patients. The treatment was recently granted rare pediatric drug designation in the U.S., a status that aims to incentivize companies to develop treatments for serious or life-threatening rare diseases that mainly affect children.. FORWARD-53 enrolled 11 boys with DMD, ages 5 to 11, all ...
Top 10 muscular dystrophy stories of 2023
网页2024年1月2日 · 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 ...
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网页MDA Engage offers neuromuscular disease updates, connections. The Muscular Dystrophy Association (MDA) is providing neuromuscular disease (NMD) patients and their families with the opportunity to meet experts and other people on similar journeys through its Engage Symposiums, free in-person events that include disease-specific sessions as well as talks focusing on NMDs in general.
MDA 2023: Exondys 51 may boost survival for DMD patients
网页2023年3月21日 · The longest survival times were generally seen among patients who started on therapy early and were treated for many years. Findings were presented this month at the MDA Clinical & Scientific Conference, in the poster “Survival in Eteplirsen-Treated vs Duchenne Muscular Dystrophy Natural History Patients: An Indirect Treatment Comparison Using Real-World Data.”
Rare pediatric disease designation granted to LAMA2-CMD therapy
网页2 天之前 · The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to MDL-101, an epigenetic editing therapy that Modalis Therapeutics is developing for LAMA2-related congenital muscular dystrophy (LAMA2-CMD) — a genetic condition that’s characterized by muscle wasting evident at birth or shortly thereafter.
LGMD type 2A mutation corrected by CRISPR-Cas9 gene editing
网页2023年3月9日 · “We show an effective and precise repair to correct the most frequent [LGMD type 2A] mutation … Our approach is a promising source for autologous [self-derived] cell replacement therapies for LGMD” type 2A, the researchers wrote in the study, “ Cas9-induced single cut enables highly efficient and template-free repair of a muscular dystrophy causing founder mutation,” which was ...
New Viral Vector, Used in MD, May Improve Gene Therapy …
网页2021年9月14日 · In fact, this new vector “is more than 10 times more efficient at reaching muscle than those currently used in clinical trials,” the scientists said in a news story.. The researchers described the new vector in the journal Cell, in a study titled “ Directed evolution of a family of AAV capsid variants enabling potent …
#MDA2021 – DMD Gene Therapy PF-06939926 Remains Promising
网页2021年3月24日 · Pfizer‘s PF-06939926 uses a harmless adeno-associated virus (AAV9) to deliver a mini-dystrophin gene, which encodes a smaller but functional version of dystrophin to muscle cells.It is designed to slow or stop muscle degeneration. The ongoing Phase 1b study (NCT03362502) is evaluating the safety and tolerability of PF-06939926 in boys who can walk at least 10 meters (about 33 feet) without ...