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The Journey from Lab to Clinic: Zolgensma, the First Gene Therapy Treatment for Spinal Muscular Atrophy (SMA)

Translational research bridges the gap between scientific discoveries and real-world applications, transforming scientific breakthroughs into life-changing therapies. Success in this endeavor hinges on several factors, including collaboration between scientists and clinicians. A notable example of this is the approval of Zolgensma, a gene therapy for spinal muscular atrophy (SMA), which marked a significant milestone in the field of translational research.
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