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August 7, 2025Annals of Indian Academy of NeurologyOpen Access

Evaluating Therapeutic Outcomes in Spinal Muscular Atrophy: An Indian Experience

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Authors

SMSmilu MohanlalPMPM MubeenaHHHuma Hussain

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Overview

Observational study shows improvements in motor function with risdiplam, nusinersen, and gene therapy in SMA patients, indicating diverse efficacy based on disease severity.

Key Points

  • Gene therapy resulted in the highest motor function improvement as measured by HFMSE, with a median increase of 4 points.
  • This study analyzed 47 SMA patients aged 1-23, assessing outcomes over 6 months using validated scales.
  • All disease-modifying therapies showed positive effects, highlighting potential personalized approaches to treatment.
  • A significant correlation was found between SMN2 copy numbers and baseline disease severity, indicating its role in treatment evaluation.

Cite This Study

Mohanlal et al. (2025) studied this question.

synapsesocial.com/papers/689dfe88d61984b91e13b946https://doi.org/10.4103/aian.aian_670_24
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Emerging Therapeutic Strategies for Spinal Muscular Atrophy: A Comprehensive Review2024
  2. 2Disease characteristics and treatment status of genetically confirmed spinal muscular atrophy patients: a cross-sectional survey in China2025
  3. 3Treatment evolution in spinal muscular atrophy: insights from the SMArtCARE registry2025 · 2 citations
  4. 4Longitudinal efficacy of risdiplam treatment in Chinese children with spinal muscular atrophy2025
  5. 5Clinical Characteristics, Genotypes, and Treatment Outcomes in 133 Patients With Spinal Muscular Atrophy: A Retrospective Cohort2025