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Treatment with antimiRs, an RNA-based therapy approach, was able to correct molecular defects in a cell model of myotonic dystrophy type 1 (DM1) — a form of muscular dystrophy — according to the results of a new study. The researchers called their work “a significant step forward,” and noted that…. October 16, 2024 Columns by Robin ...
MDA 2024: Gene therapy’s benefits most pronounced in young boys
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 ...
Top 10 muscular dystrophy stories of 2023
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 ...
News Archives - Muscular Dystrophy News
Treatment with antimiRs, an RNA-based therapy approach, was able to correct molecular defects in a cell model of myotonic dystrophy type 1 (DM1) — a form of muscular dystrophy — according to the results of a new study. The researchers called their work “a significant step forward,” and noted that…. October 15, 2024 News by Lindsey ...
MDA 2024: Limb, heart function preserved with DMD cell therapy
2024年3月14日 · The investigational cell therapy CAP-1002 has continued to slow declines in upper limb and heart function among boys and young men with Duchenne muscular dystrophy (DMD), according to two-year results from the HOPE-2 open label extension (OLE) study. The findings were presented at the recent 2024 Muscular Dystrophy Association (MDA) Clinical ...
MDA 2023: Exondys 51 may boost survival for DMD patients
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 …
Top 10 MD Stories of 2021 - Muscular Dystrophy News
2022年1月4日 · Audentes Therapeutics, CIFFREO Phase 3 clinical trial, EMBARK clinical trial, Fulcrum Therapeutics, gene therapies, PF-06939926, Pfizer, Roche, Sarepta Therapeutics, SRP-9001, Top 10. Here are the top 10 most-read articles of 2021, with a brief description of what made them relevant for the MD community.
Top 10 Most-read Muscular Dystrophy Stories of 2020
2021年1月5日 · Bionano Genomics, DMD gene, Duchenne muscular dystrophy, Dystrophin, Fulcrum Therapeutics, Gene Therapy, Hansa Biopharma, imlifidase, losmapimod, micro-dystrophin, PerkinElmer Genomics, PF-06939926, Pfizer, Sarepta Therapeutics, Top 10. Check out the top 10 muscular dystrophy stories of 2020, which included coverage of …
#MDA2021 – DMD Gene Therapy PF-06939926 Remains Promising
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 ...
MDA 2022 Archives - Muscular Dystrophy News
2022年3月15日 · The corticosteroid Emflaza outperforms prednisone at slowing disease progression in people with Duchenne muscular dystrophy (DMD), according to new research presented at the Muscular Dystrophy Association (MDA) 2022 Annual Meeting being held this week in Nashville, Tennessee. The research was funded by …