Effective Strategies for Managing Spinal Muscular Atrophy
This article explores available treatments and supportive strategies for managing spinal muscular atrophy. It highlights FDA-approved medications like Spinraza, Zolgensma, and Risdiplam, along with assistive devices and physical therapies that improve quality of life based on current medical research and clinical practice.
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Spinal muscular atrophy (SMA) is a neurological disorder characterized by progressive muscle weakness caused by the degeneration of motor neurons in the spinal cord and brainstem. While no definitive cure exists, various treatments can significantly improve quality of life and help manage symptoms.
Approved Medications
Research continues into SMA therapies, and a few medications have gained FDA approval:
Nusinersen (Spinraza)
This drug is designed for both children and adults with SMA. It involves an initial series of four injections over two months, followed by a maintenance dose every four months. Nusinersen modifies the SMN2 gene to produce more functional protein, potentially slowing disease progression and reducing muscle weakness.
Zolgensma (Onasemnogene abeparvovec-xioi)
Ideal for children under two, this gene therapy is administered as a one-time infusion through a vein. It introduces a functional copy of the SMN gene, helping infants reach developmental milestones like holding their heads up and sitting independently.
Evrysdi (Risdiplam)
Taken orally once daily, Risdiplam aims to enhance SMN protein production. Clinical trials indicated that about 41% of patients experienced improved muscle function after a year of treatment.
Supportive Devices and Therapeutic Approaches
Complementary treatments, such as assistive devices—ventilators, powered wheelchairs, and adaptive computer access—help individuals with SMA stay active and involved. Physical therapies, including aquatic exercises and wheelchair sports, further aid in maintaining mobility and enhancing overall well-being.
