Lipodystrophy due to peptidic growth factors deficiency
MONDO:0009312Deficiency of the peptidic growth factors is characterized by loss of subcutaneous fat layers on the limbs, lipodystrophy in the face and trunk and scleroderma-like skin disorders (thickened skin on the palms and soles and skin pigment changes on the limbs and trunk).
Also known as: Hoepffner-Dreyer-Reimers syndrome, Werner-like syndrome due to combined growth factor deficiency, combined insulin, insulin-like growth factor 1 (IGF1) and epidermal growth factor (EGF) deficiency, Hoepffner Dreyer Reimers syndrome, Werner-like syndrome due to combined Growth Factor deficiency, growth factors, combined defect OF, insulin, insulin-like Growth Factor I, and Epidermal Growth Factor deficiency, peptide growth factors deficiency
3 clinical trials for this condition and its sub-types.
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Rare leptin disorder patients gain access to experimental drug
Disease control AVAILABLEThis program provides expanded access to REGN4461 (mibavademab) for people with rare diseases caused by deficient leptin signaling, such as generalized lipodystrophy and monogenic obesity. The drug aims to help control metabolic issues linked to these conditions. Participants rec…
Sponsor: Regeneron Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC
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Massive european registry launches to unlock secrets of rare fat disorder
Knowledge-focused Recruiting nowThis study is a European registry for people with lipodystrophy, a rare condition where the body loses or lacks fat tissue. Researchers will follow up to 5,000 patients over time, collecting health data and genetic information. The goal is to better understand the disease, its pr…
Sponsor: University of Ulm • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:01 UTC