FDA Approves First Gene Therapy for Treatment of Certain …
2023年6月23日 · Today, the U.S. Food and Drug Administration approved Elevidys, the first gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy (DMD)...
FDA Expands Approval of Gene Therapy for Patients with …
2024年6月20日 · Today, the U.S. Food and Drug Administration expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) for...
AAV gene therapy for Duchenne muscular dystrophy: the …
2024年10月9日 · A gene transfer therapy study to evaluate the safety and efficacy of delandistrogene moxeparvovec (SRP-9001) following imlifidase infusion in participants with Duchenne muscular dystrophy...
AAV gene therapy for Duchenne muscular dystrophy: the …
Delandistrogene moxeparvovec, an adeno-associated rh74-viral vector-based gene therapy, addresses absent functional dystrophin in DMD. Here the phase 3 EMBARK study aimed to assess the efficacy and safety of delandistrogene moxeparvovec in patients with DMD.
The FDA and Gene Therapy for Duchenne Muscular Dystrophy
The FDA is evaluating clinical trial results that will inform whether the gene therapy for DMD becomes available to more children diagnosed with this progressive, irreversible, and ultimately fatal rare disease.
Gene Therapy for Duchenne Muscular Dystrophy - PMC
2021年11月30日 · MICRO-DYSTROPHIN GENE THERAPY: SHRINKING THE DMD GENE. The mRNA ORF coding sequence of the DMD gene is some 11.5 kb long and this large size poses a huge challenge in devising gene transfer therapies .
Therapeutic approaches for Duchenne muscular dystrophy
2023年8月31日 · Classical gene therapy for DMD aims to introduce DNA that encodes a functional dystrophin protein into patient muscles. Transgene DNA is typically delivered using viral vectors, with AAV...
FDA approves first gene therapy for Duchenne muscular dystrophy ...
2023年6月23日 · The FDA has granted accelerated approval to Sarepta’s gene therapy delandistrogene moxeparvovec (Elevidys) for Duchenne muscular dystrophy (DMD). It is the first time the FDA has...
Muscular dystrophy patients get first gene therapy - NPR
2023年6月22日 · The Food and Drug Administration approved the first gene therapy for Duchenne muscular dystrophy, but limited access to those ages four and five.
Gene Therapy Details Revealed in Duchenne Trial - Medpage Today
4 天之前 · by Judy George, Deputy Managing Editor, MedPage Today October 18, 2024. SAVANNAH, Ga. -- Delandistrogene moxeparvovec (Elevidys), a gene therapy approved to treat Duchenne muscular dystrophy ...