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Real-world data fill knowledge gap to assess treatment options for infants with spinal muscular atrophy

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Findings from a recent study in the Journal of Neuromuscular Diseases demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy (SMA). The study's results add further support for gene therapy as a treatment modality that can deliver durable transformative effects for these vulnerable patients.

Real-world data fill knowledge gap to assess treatment options for infants with spinal muscular atrophy retrieved 15 April 2024 from https://medicalxpress.com/news/2024-04-real-world-knowledge-gap-treatment.html

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