N-Acetyl Cysteine and Improved Motor Function

• A Randomized, Double-Blind, Pilot Study of N-Acetylcysteine for Motor and Cognitive Symptoms in Youth With Neurofibromatosis Type 1.

  • 2026-07
  • RCT · n = 481
  • Pediatric neurology 180
  • • Donald L Gilbert
    • Lindsey E Aschbacher-Smith
    • Karlee Y Migneault
    • David A Huddleston
    • Kim M Cecil
    • Dean W Beebe
    • Bin Zhang
    • Nancy Ratner
    • Carlos E Prada

  • PubMed: 42173046
  • DOI: 10.1016/j.pediatrneurol.2026.04.024

  3 Insights on

  • Improved Motor Function

  • High evidence
  • Large Human Trial
  • Clinical

  no significant difference in Physical and Neurological Examination for Subtle Signs was observed between groups

  Effect

  Neutral

  Effect size

  Small

  Significant

  No

  Dose

  ~70 mg/kg/day