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
- High evidence
- Large Human Trial
- Clinical
NAC did not improve any secondary or exploratory outcome at 8 weeks
- Effect
- Neutral
- Effect size
- Small
- Significant
- No
- Dose
- ~70 mg/kg/day