Huntington's disease (HD) is a neurodegenerative disease with cognitive, motor and psychiatric symptoms. Toxic accumulation of misfolded mutant huntingtin protein induces mitochondrial dysfunction, leading to a bioenergetic insufficiency in neuronal and muscle cells. We evaluated the safety, pharmacokinetics and pharmacodynamics of SBT-020, a novel compound to improve mitochondrial function, in a 2-part study in early stage HD patients.
Safety, pharmacokinetics and pharmacodynamics of SBT-020 in patients with early stage Huntington's disease, a 2-part study.
CHDR
van Diemen MPJ, Hart EP, Abbruscato A, Mead L, van Beelen I, Bergheanu SC, Hameeteman PW, Coppen E, Winder JY, Moerland M, Kan H, van der Grond J, Webb A, Roos RAC, Groeneveld GJ
