PPARG-related familial partial lipodystrophy
MONDO:0011448Also known as: FPLD3, PPARG-related FPLD, familial partial lipodystrophy type 3, familial partial lipodystrophy associated with PPARG mutations, insulin resistance, severe, digenic, lipodystrophy, familial partial, associated with Pparg mutations, lipodystrophy, familial partial, type 3
5 clinical trials for this condition and its sub-types, 0 tagged with PPARG-related familial partial lipodystrophy itself.
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Rare leptin disorder patients gain access to experimental drug
Disease control Expanded accessThis 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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800-Patient registry maps the hidden path of rare muscle and heart gene disorders
Knowledge-focused Recruiting nowLaminopathies and emerinopathies are rare disorders caused by mutations in the LMNA or EMD genes, often affecting skeletal muscles and the heart. Researchers at French hospitals are building a registry of 800 patients with confirmed mutations to record detailed genetic, neurologi…
Sponsor: Pitié-Salpêtrière Hospital • Aim: Knowledge-focused
Last updated Sep 21, 2026 16:00 UTC
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Scientists hunt for 'Modifier Genes' that could explain why some LMNA patients fare better than others
Knowledge-focused Recruiting nowThis study aims to identify genetic factors that affect how severe muscle and heart problems become in people with LMNA gene mutations. Researchers will collect skin and muscle samples from 40 participants and use advanced DNA and RNA analysis to look for protective or aggravatin…
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:10 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