Gene therapy SRP-9003 for beta-myolysis achieves positive results in Phase 1/2a clinical trials
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Last Update: 2020-06-08
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Source: Internet
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Author: User
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Recently, Sarepta Therapeutics(http://announced positive results in its gene therapy SRP-9003 (MYO-101) for the treatment of beta-myolysis (LGMD2E) in the(http://of the 1/2a ClinicalTrialSRP-9003 not only improves the indicators for evaluating muscle function in patients, but also significantly reduces the level of serum creatine kinase (CK), an important biomarker associated with muscle damage in patientson SRP-9003
SRP-9003 is a gene therapy developed by Myonexus to treat LGMD2EIn 2018, a research and development cooperation agreement was concluded with Sarepta to conduct a clinical trial of SRP-9003In patients with LGMD2E, a mutation in the gene that expresses beta-myolysis causes the loss of beta-myolysis, which causes muscle fiber loss, inflammation, and muscle fiber to be replaced by fat and fibrosis scar tissueSRP-9003 by using adeno-related virus (AAV) vector, the normal gene of expression beta-myolysis is introduced into the patient's heart muscle and skeletal muscle, thereby increasing beta-myolysis levels and preventing or even reversing the progression of the diseaseIn the first queue of the clinical trial, three patients aged 4-13 with LGMD2E were treated with SRP-9003The results showed that after 9 months of treatment, patients had improved their time on muscle function indicators such as muscular dystrophy assessment (NSAD), up, fourth-order climb, 100-meter walking, and 10-meter walking, after 9 months of treatment
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