NIIGATA UNIVERSITY

L-Arginine Shows Promise but Falls Short of Statistical Significance in Spinocerebellar Ataxia Type 6 Trial

- A Phase 2 trial of L-arginine in Spinocerebellar Ataxia type 6 (SCA6) showed a trend toward symptom improvement, but the results did not reach statistical significance. - The L-arginine group experienced a 0.96-point improvement in SARA scores, while the placebo group worsened by 0.56 points over 48 weeks. - Two serious adverse events potentially linked to L-arginine were reported, including one fatality due to pneumonia and one case of reversible liver impairment. - Further Phase 3 trials with larger cohorts are needed to confirm L-arginine's efficacy and safety in treating SCA6, a neurodegenerative disorder.