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Study · Case report or series · n = 2 · 2025

1 result

Efficacy of Semaglutide in Pediatric Patients With Bardet-Biedl Syndrome and Alström Syndrome.

Dauleh H, Mohammed I, Hussain K

JCEM case reports26 Nov 2025Cited by 3 (Europe PMC)

PMID41312179 (opens in a new tab)PMCIDPMC12647937 (opens in a new tab)DOI10.1210/jcemcr/luaf266 (opens in a new tab)PepBankPUB-41312179
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Summary

Bardet-Biedl syndrome (BBS) and Alström syndrome (AS) are rare autosomal recessive ciliopathies characterized by severe multisystemic involvement, including metabolic, sensory, and developmental impairments. Both conditions result from primary cilia dysfunction, disrupting pathways such as leptin and insulin signaling, which leads to obesity and insulin resistance. While setmelanotide, a melanocortin-4 receptor agonist, may be an effective therapy for obesity in these conditions (especially BBS), its cost limits accessibility for many patients. We describe two pediatric cases (one with BBS and another with AS) demonstrating significant metabolic improvements with semaglutide, a glucagon-like peptide-1 receptor agonist. These observations suggest that some cases of BBS and AS may be treated with semaglutide.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study1

1 result from studies that gave the peptide. 1 result from people.

  1. 01HumanCase report or seriesEfficacy outcome

    In two children with Bardet-Biedl syndrome or Alström syndrome, semaglutide was reported to give significant metabolic improvements.

    In plain wordsIn two children with rare genetic obesity syndromes, doctors reported clear metabolic improvements while the children took semaglutide.

    Peptide
    Semaglutide
    Outcome
    Direction not reportedMetabolic improvement
    Study

    n = 2

    Outcome group
    Cardiometabolic risk factors · Metabolic and weight
    Studied dose
    Dose not stated

    Source sentences

    Summary
    We describe two pediatric cases (one with BBS and another with AS) demonstrating significant metabolic improvements with semaglutide, a glucagon-like peptide-1 receptor agonist.

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

  1. Version 3Current

    27 Oct 2026, 16:36 UTC

    Study data updated · source update from Europe PMC

  2. Version 2

    11 Oct 2026, 00:04 UTC

    Study results updated

  3. Version 1

    9 Oct 2026, 03:06 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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