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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, 1 tagged with Severe early-onset obesity-insulin resistance syndrome due to SH2B1 deficiency itself.

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Part of

↑ Hereditary disease (18187) ↑ Syndromic disease (7133)

Including sub-types (1) Tagged with Severe early-onset obesity-insulin resistance syndrome due to SH2B1 deficiency (1)

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 3 • Sponsor: Rhythm Pharmaceuticals, Inc. • Aim: Disease control

Last updated Aug 08, 2026 00:03 UTC