WebApr 11, 2024 · Currently, the only approved gene therapy for the treatment of spinal muscular atrophy (SMA) is onasemnogene abeparvovec (Zolgensma; Novartis), indicated … WebGARD: 19 Spinal muscular atrophy (SMA) is a group of genetic neuromuscular disorders that affect the nerve cells that control voluntary muscles (motor neurons). The loss of motor neurons causes progressive muscle weakness and loss of movement due to muscle wasting (atrophy).
Single-Dose Gene-Replacement Therapy for Spinal …
WebThe Spinal Muscular Atrophy Clinic, along with the Center for Gene Therapy in The Research Institute at Nationwide Children’s Hospital and the OSU Division of Neuromuscular Medicine, is actively involved in clinical trials for children and adults with SMA. Our Clinical Research Unit is the only center in the world that has carried out ... WebA phase 1 clinical trial of SRK-015 in healthy volunteers resulted in no adverse effects, successful inhibition of myostatin, and a relatively long half-life. Its success supported the advancement toward a phase 2 clinical trial. BIIB110, developed by Biogen, is a hybrid inhibitor that acts on both myostatin and activins, but not on BMP9. swot time
Observational study of spinal muscular atrophy type I and ... - PubMed
WebDecember 23, 2016. The U.S. Food and Drug Administration today approved Spinraza (nusinersen), the first drug approved to treat children and adults with spinal muscular … WebPhase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients With Later-Onset Spinal Muscular Atrophy Receiving … WebAlthough more work is needed to fully understand the mechanism of SMA disease pathogenesis, promising targets for SMA therapeutics are identified and early clinical trials of compounds directed to these targets are ongoing in SMA patients. AB - Spinal muscular atrophy (SMA) is a currently untreatable, autosomal recessive motor neuron disease. text flex wrap