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PPARG-associated congenital generalized lipodystrophy

MONDO:0979300

3 clinical trials for this condition and its sub-types.

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Broader categories

Disease (680) Metabolic disease (233) Hereditary disease (176) Skin disorder (132) Inborn errors of metabolism (45) Lipodystrophy (27) Human disease (14) Hereditary skin disorder (6) Disease of genetic or genomic mechanism (2) Hereditary lipodystrophy (2)
Trials to join now! 2 Completed 1
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  • Rare leptin disorder patients gain access to experimental drug

    Disease control AVAILABLE

    This 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

  • Massive european registry launches to unlock secrets of rare fat disorder

    Knowledge-focused Recruiting now

    This 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

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