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August 14, 2025Brain and DevelopmentOpen Access

Survival motor neuron protein is the optimal biomarker for evaluating the risdiplam treatment

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Authors

TKTamaki KatoNONoriko OtsukiMYMamoru Yokomura

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Overview

Clinical trial evaluates risdiplam's effectiveness in spinal muscular atrophy using survival motor neuron protein as a biomarker.

Key Points

  • Survival motor neuron protein levels significantly increased after one month of risdiplam treatment, indicating treatment responsiveness.
  • Hammersmith Functional Motor Scale and Revised Upper Limb Module scores showed significant improvements at months 5, 8, and 12, supporting motor function enhancement.
  • Observational cohort study assessed 25 patients over 12 months, indicating promising outcomes with risdiplam for spinal muscular atrophy.
  • Use of survival motor neuron protein as a biomarker highlights the need for precise monitoring of therapeutic efficacy in clinical settings.

Cite This Study

Kato et al. (2025) studied this question.

synapsesocial.com/papers/68a34f4c234c60ad5c20b7bahttps://doi.org/10.1016/j.braindev.2025.104410
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Also Consider

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

  1. 1Longitudinal efficacy of risdiplam treatment in Chinese children with spinal muscular atrophy2025
  2. 2Safety of Risdiplam in Japanese Patients with Spinal Muscular Atrophy: A 12‑Month Interim Analysis of a Postmarketing Surveillance Study2025 · 1 citations
  3. 3Risdiplam in Presymptomatic Spinal Muscular Atrophy2025 · 38 citations
  4. 4Evaluating Therapeutic Outcomes in Spinal Muscular Atrophy: An Indian Experience2025 · 1 citations
  5. 5Neurofilaments as Biomarkers of the Efficacy of Risdiplam Treatment in Early SMA Phenotypes Diagnosed by Newborn Screening2025