New hope for youngest kids with rare fat disorder
Disease control
Terminated
This study tests a drug called metreleptin in 15 children under 6 years old who have a rare disease called generalized lipodystrophy, which prevents the body from storing fat properly and often leads to diabetes and high triglycerides. The goal is to see if the drug can lower blo…
Phase: PHASE3 • Sponsor: Amryt Pharma • Aim: Disease control
Last updated Jul 24, 2026 00:00 UTC