The RAINBOW study: a phase 2 trial evaluating the 3-month pharmacokinetic and pharmacodynamic data and 18-month clinical and safety outcomes of nizubaglustat in GM2 gangliosidoses or Niemann-Pick type C disease.

nizubaglustat (AZ-3102) - Azafaros

http://www.ncbi.nlm.nih.gov/pubmed/42308595

Aidan Gill; Dafne D G Horovitz; Daniel Almeida do Valle; Laura López de Frutos; Mariana Horn Scherer; Michelle Zeny; Roberta Tuche; Roberto Giugliani; Tamires Silva Alves

Jun 18, 2026