Spinal Muscular Atrophy (SMA) is a genetic disorder characterized by the degeneration of motor neurons, leading to muscle weakness and atrophy. Recent breakthroughs in treatment have transformed the landscape for SMA patients. The advent of gene therapy, particularly with the FDA-approved drug Zolgensma, has revolutionized care by targeting the root cause of the disease. Zolgensma delivers a functional copy of the SMN1 gene, significantly improving muscle function and survival rates in infants diagnosed with the condition.
Additionally, other therapies like Nusinersen (Spinraza) and Risdiplam (Evrysdi) have provided options that enhance spinal motor neuron health and increase SMN protein levels. These treatments represent a paradigm shift, moving SMA care towards proactive management rather than reactive measures. Rehabilitation and supportive therapies also play a crucial role, focusing on enhancing quality of life. Education on these advancements empowers families and patients, providing hope for a brighter future in managing SMA.
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