Thomas Payne, Matthew Appleby, Ellen Buckley, Linda M A van Gelder, Benjamin H Mullish, Matilde Sassani, Mark J Dunning, Dena Hernandez, Sonja W Scholz, Alisdair McNeill, Vincenzo Libri, Sarah Moll, Julian R Marchesi, Rosie Taylor, Li Su, Claudia Mazzà, Thomas M Jenkins, Thomas Foltynie, Oliver Bandmann
BACKGROUND: Rescue of mitochondrial function is a promising neuroprotective strategy for Parkinson's disease (PD). Ursodeoxycholic acid (UDCA) has shown considerable promise as a mitochondrial rescue agent across a range of preclinical in vitro and in vivo models of PD. OBJECTIVES: To investigate the safety and tolerability of high-dose UDCA in PD and determine midbrain target engagement. METHODS: The UP (UDCA in PD) study was a phase II, randomized, double-blind, placebo-controlled trial of UDCA (30 mg/kg daily, 2:1 randomization UDCA vs...
May 29, 2023: Movement Disorders: Official Journal of the Movement Disorder Society