GM2 gangliosidosis Clinical Trial

Azafaros announcement

Azafaros announces enrollment of first patient in Phase 2 RAINBOW Study evaluating AZ-3102 in GM2 gangliosidosis and NP-C Patients. RAINBOW study design enables rapid advancement into the company’s planned Phase 3 efficacy trials.

The clinical trial (NCT05758922) is being conducted in Brazil and the US.

Press Release:

Alex - The Leukodystrophy Charity