New registry aims to unlock secrets of rare genetic diseases
Knowledge-focused
Recruiting now
This observational registry is collecting information from up to 1,000 people with ENPP1 deficiency or infantile-onset ABCC6 deficiency. The goal is to understand how these rare diseases progress over time by tracking genetic, physical, and quality-of-life changes during routine …
Sponsor: Inozyme Pharma • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:06 UTC