Global study tracks rare muscle disease to guide future care
Knowledge-focused
Terminated
This study is an international patient registry designed to collect information on how people with Lipin-1 deficiency are managed and treated. It aims to understand survival rates, heart function, quality of life, and how well different treatments work by observing patients over …
Sponsor: Imagine Institute • Aim: Knowledge-focused
Last updated Apr 01, 2026 14:42 UTC