Zolgensma Gene Therapy for SMA
Summary: Zolgensma, a costly gene therapy for Spinal Muscular Atrophy (SMA), offers hope to affected families in India by replacing the defective SMN1 gene to improve motor function. SMA is a rare disorder causing severe muscle weakness and loss, often apparent in infancy. Awareness and early diagnosis are essential for better outcomes, though treatment costs and limited access present significant challenges. Many families rely on crowdfunding and community support for this life-saving drug, while ongoing research seeks to lower costs and improve access.
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