Exciting Advances in Azafaros Clinical Programs for NPC and GM Gangliosidoses
Today, Azafaros shared an important update about their groundbreaking efforts to develop treatments for Niemann-Pick Disease Type C (NPC). Their Phase 2 RAINBOW trial for the investigational therapy nizubaglustat has shown promising safety and clinical data, with plans for a global Phase 3 trial to commence in mid-2025.
Additionally, findings from the PRONTO natural history study are helping shape future research and offer critical insights into NPC and GM gangliosidoses.
We are excited to share this progress with our community and encourage you to read the full letter now available for download below.
Let’s keep building momentum towards better outcomes!
To join our fight, as well as fund vital research for a cure, support the Australian NPC Disease Foundation in its goals of "Research. Cure. Persevere."
Ways to support NP-C in Australia:
Shop at our online NPC store
Fundraise your way! Check our free resources and inspo online.