Pharmacy and Wellness Review
Abstract
Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder characterized by the progressive loss of alpha motor neurons in the anterior horn of the spinal cord, leading to muscle weakness, atrophy, and impaired motor function.1 SMA remains an extensive and difficult-to-treat condition, with only three disease-modifying therapies currently available to patients. A new investigational agent with a unique mechanism of action, apitegromab (SRK-015), has shown promising results in recent clinical trials. The safety and efficacy of apitegromab in patients with SMA have been explored in several clinical trials, with the TOPAZ and SAPPHIRE studies being the most notable. This review summarizes the pathology, diagnostic considerations, and clinical subtypes of SMA, and evaluates the mechanism, clinical evidence, and emerging therapeutic role of apitegromab in the modern treatment landscape.
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Medical Pharmacology Commons, Medicinal and Pharmaceutical Chemistry Commons, Musculoskeletal Diseases Commons, Musculoskeletal, Neural, and Ocular Physiology Commons, Pharmaceutical Preparations Commons, Pharmaceutics and Drug Design Commons