Amryt Pharma

This study is testing a drug called metreleptin in children under 6 years old who have a rare condition called generalized lipodystrophy, which causes severe problems with fat storage, diabetes, and high blood fats. The main goal is to see if the drug safely lowers blood sugar an…

This study is a long-term follow-up for people who completed a previous trial of metreleptin, a hormone treatment given as a daily injection under the skin. It aims to monitor the safety and continued effectiveness of the treatment over time in 24 participants with familial parti…

This study is creating a registry to monitor patients who are already taking the drug metreleptin for lipodystrophy, a rare disorder affecting body fat. The goal is to collect long-term safety information on side effects and how well the treatment works in everyday medical practi…

This study aims to understand if using the topical gel Filsuvez affects the risk of developing skin cancers in people with severe forms of Epidermolysis Bullosa (EB), a condition that causes extremely fragile, blistering skin. Researchers will follow about 580 patients with EB fo…