First patient dosed in global pivotal clinical trial program for late infantile/juvenile onset NPC and GM1/GM2 gangliosidoses
The Phase 3 program consists of two studies targeting the late-infantile and juvenile-onset forms of NPC, and GM1/GM2 gangliosidoses. The studies aim to assess the potential of nizubaglustat to alter disease progression and improve functional outcomes in these patient populations.
To read full press release: Azafaros press release
