Amryt Pharma

This study looks at the long-term safety and effectiveness of a daily injection called metreleptin for people with familial partial lipodystrophy, a rare condition where fat is distributed abnormally. About 24 participants who completed a previous study will receive the drug for …

This registry monitors the safety and effectiveness of metreleptin in up to 100 people with generalized or partial lipodystrophy who are already taking the drug in routine care. Researchers will track serious events like pancreatitis, liver problems, infections, and cancers. The …

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…

This study follows 580 people with dystrophic or junctional epidermolysis bullosa (EB) for up to 5 years. Researchers want to see if using Filsuvez, a birch bark extract gel that helps heal blisters, changes the risk of developing skin cancer. Participants receive standard care, …