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Efficacy of Metreleptin Treatment in Familial Partial Lipodystrophy Due to PPARG vs LMNA Pathogenic Variants

CONTEXT: Familial partial lipodystrophy (FPLD) is most commonly caused by pathogenic variants in LMNA and PPARG. Leptin replacement with metreleptin has largely been studied in the LMNA group. OBJECTIVE: To understand the efficacy of metreleptin in PPARG vs LMNA pathogenic variants and investigate p...

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Pubblicato in:J Clin Endocrinol Metab
Autori principali: Sekizkardes, Hilal, Cochran, Elaine, Malandrino, Noemi, Garg, Abhimanyu, Brown, Rebecca J
Natura: Artigo
Lingua:Inglês
Pubblicazione: Endocrine Society 2019
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6563798/
https://ncbi.nlm.nih.gov/pubmed/31194872
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1210/jc.2018-02787
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