Community News

Get the latest news from NTSAD about our Community, the research that provides hope, and what is happening in the world of rare disease.

Latest Issue – July 2023

In this issue:

  • Azafaros GM2 Clinical Trial
  • New Directory of Patient Samples
  • Working for Noa: Canavan Gene Therapy
  • Join Us in a Day of Hope
  • The Manning Family: 20 Years of Honoring Dylan
  • Stephanie Wright: Living with Late Onset Disease
  • Why I Give to The Buryk Research Fund
  • Imagine & Believe 2023
  • Gottlieb Scholarship for Rare Siblings


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Azafaros Recruiting for GM2 Clinical Trial  

Azafaros, a biotech company based in the Netherlands, began recruiting for its RAINBOW Study, a phase 2 multinational trial for Tay-Sachs, Sandhoff, (known collectively as GM2) and Niemann-Pick Type C. The study will be held in Brazil and the United States.

Recently, NTSAD and the National Niemann-Pick Disease Foundation hosted a community meeting with Azafaros about the RAINBOW study and answered questions from rare families. The Phase 2 RAINBOW study is a randomized, double-blind, placebo-controlled, multicenter, 12-week trial assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of AZ-3102 in patients with GM2 gangliosidosis and NP-C. The study will enroll 12 patients in total (six with GM2 and six with NP-C), aged 12 to 20 years, each of whom will receive either the low dose, high dose, or a placebo. Patients who complete the 12-week study period will be offered a double-blind extension if approved by the country’s health authorities. The aim of the short study is to determine the clearance of AZ-3102 from the body and the effect of two different doses in patients to identify the target dose for Azafaros’ planned Phase 3 pivotal studies.

Watch the community meeting with Azafaros about the RAINBOW Study, now available on NTSAD’s YouTube channel.