Azafaros to present data from PRONTO study in patients with GM1 and GM2 gangliosidoses at the 20th annual WORLDSymposium™
As well as data from PRONTO, Azafaros will present details of its Phase 2 RAINBOW study, aimed at investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of nizubaglustat in patients with GM2 gangliosidosis or NPC.
- As well as data from PRONTO, Azafaros will present details of its Phase 2 RAINBOW study, aimed at investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of nizubaglustat in patients with GM2 gangliosidosis or NPC.
- Title: A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO) - patients’ and caregivers’ assessments.
- Title: A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO)- baseline clinical data.
- Title: A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO) - evaluation of different assessments.