2 results
Approved WMOCompleted
The primary objective of TRIHEP 3 is to evaluate the efficacy of triheptanoin in (i) increasing the short term energy response in the metabolic profile of the brain of early affected HD patients, as captured by 31P-MRS, and (ii) slowing atrophy in…
Approved WMOPending
Primary:To assess the safety and tolerability of DS-1211b compared with placebo in subjects with PXE.To assess the dose response by assessing the treatment changes in PD endpoints.