3 results
To determine RD of BLU-263To assess if treatment with BLU-263 improves outcomes compared to placebo + BSC, as assessed using the ISM-SAFTo assess the long-term safety and tolerability of treatment with BLU-263To assess the long-term efficacy of…
The primary objective is to compare the effectiveness of ambroxol versus placebo on the MDS-UPDRS part III motor sub-score in the *practically defined OFF-medication state* in patients with moderate PD, carrying a GBA mutation. The hypothesis is…
The current study is designed to evaluate the preliminary safety and efficacy of BLU-263 in patients with AdvSM, including in those with high and very high-risk SM-AHN, in whom HMAs, and azacitidine specifically, are the standard of care. Dose…