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Severe early-onset obesity-insulin resistance syndrome due to SH2B1 deficiency

MONDO:0017994

1 clinical trial for this condition and its sub-types.

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Broader categories

Disease (679) Hereditary disease (176) Syndromic disease (25) Human disease (14) Disease of genetic or genomic mechanism (2) Disease by body system or component (0) Disease by etiologic mechanism (0)

Gene-Targeted obesity drug shows promise in phase 3 trial

Disease control Completed

This phase 3 trial is testing a drug called setmelanotide in people with obesity linked to certain gene variants (POMC, PCSK1, LEPR, NCOA1, or SH2B1). About 296 participants, aged 6 to 65, will receive either the drug or a placebo to see if it helps reduce body weight. The study …

Phase: PHASE3 • Sponsor: Rhythm Pharmaceuticals, Inc. • Aim: Disease control

Last updated Aug 08, 2026 00:03 UTC