New gene therapy approach shows promise for Duchenne muscular dystrophy
Indiana University researchers, led by Renzhi Han, developed a novel gene therapy using a triple-adeno-associated virus vector system to restore full-length dystrophin protein in Duchenne muscular dystrophy (DMD) mice models, improving muscle health and strength. This approach, promising better outcomes than current treatments, is advancing toward clinical development.
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SRP-9001 (Elevidys) received FDA approval for Duchenne muscular dystrophy (DMD) patients, including nonambulatory ones, with continued approval contingent on clinical benefit verification. CureDuchenne hosted webinars discussing this gene therapy's impact and future developments in DMD treatment.
The FDA expanded approval of delandistrogene moxeparvovec-rokl (Elevidys) for Duchenne muscular dystrophy (DMD) to include ambulatory and non-ambulatory individuals aged 4 and older with a confirmed DMD gene mutation. This gene therapy, producing a micro-dystrophin protein, addresses the urgent treatment need for DMD, a life-threatening disease. Despite its potential, the high cost of gene therapies poses a significant barrier to access.
FDA granted traditional approval for Sarepta Therapeutics’ SRP-9001 (Elevidys) for ambulatory Duchenne muscular dystrophy (DMD) patients aged 4+, expanding its use. Accelerated approval for nonambulatory patients was also announced, pending confirmatory trial results. SRP-9001, a gene therapy, showed functional improvements in trials, despite not meeting the primary endpoint in the phase 3 EMBARK study.
Indiana University researchers, led by Renzhi Han, developed a novel gene therapy using a triple-adeno-associated virus vector system to restore full-length dystrophin protein in Duchenne muscular dystrophy (DMD) mice models, improving muscle health and strength. This approach, promising better outcomes than current treatments, is advancing toward clinical development.
FDA approves Elevidys, a gene therapy for Duchenne muscular dystrophy, for most patients with a specific genetic mutation. Costing $3.2M per patient, it's a one-time treatment offering hope despite risks like liver injury. It marks significant progress in treating this progressive, muscle-wasting disease.