未知生产厂商
Delandistrogene moxeparvovec is a gene therapy used to treat Duchenne Muscular Dystrophy in ambulatory children with DMD gene mutation.
Delandistrogene moxeparvovec is a gene therapy used to treat Duchenne Muscular Dystrophy in ambulatory children with DMD gene mutation.
DMD is a progressive, fatal X-linked recessive disease caused by mutations in the DMD gene that encodes dystrophin, an essential protein involved in muscle integrity and function. As a component of the dystrophin-glycoprotein complex (DGC), which absorbs shock and maintains muscle integrity during normal muscle contraction, dystrophin links the intracellular cytoskeleton network of muscle fibre cells to the sarcolemma. A lack of functional dystrophin protein results in the failure of DGC assembly, muscle inflammation and damage, impaired muscle fibre regeneration, and progressive and irreversible deterioration of muscle function and mass. Delandistrogene moxeparvovec carries a transgene encoding microdystrophin. Microdystrophin is a shortened form of dystrophin and contains selected domains of dystrophin expressed in normal muscle cells. Microdystrophin delivered by delandistrogene moxeparvovec has been demonstrated to localize to the sarcolemma.
Under accelerated approval, delandistrogene moxeparvovec is indicated for the treatment of ambulatory pediatric patients four to five years old with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. This indication is approved under accelerated approval based on the expression of delandistrogene moxeparvovec microdystrophin in skeletal muscle observed in patients treated with this therapy. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
共 1 项相关疾病
