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<p>The advent of highly effective anti-obesity pharmacotherapy has transformed obesity management, yet patients with rare genetic syndromes associated with obesity remain largely excluded from evidence generation and clinical guidance. Conditions such as Turner syndrome, Klinefelter syndrome, Fragile X syndrome, and sex chromosome mosaicisms may represent relevant candidates for anti-obesity treatment, although dedicated studies are lacking. This evidence gap may contribute to therapeutic inertia and limit equitable access to therapeutic innovation. We highlight the need for dedicated studies, real-world evidence, and expert recommendations to ensure that rare diseases, already orphan conditions by definition, do not become orphan indications in the era of modern obesity pharmacotherapy.</p>

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Beyond monogenic obesity: the unmet need for anti-obesity pharmacotherapy in rare genetic syndromes

  • 期刊论文

  • 发表日期：

    2026年7月23日

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  • Angelo Di Vincenzo,
  • Marnie Granzotto,
  • Elisabetta Trevellin,
  • Marco Rossato

摘要

The advent of highly effective anti-obesity pharmacotherapy has transformed obesity management, yet patients with rare genetic syndromes associated with obesity remain largely excluded from evidence generation and clinical guidance. Conditions such as Turner syndrome, Klinefelter syndrome, Fragile X syndrome, and sex chromosome mosaicisms may represent relevant candidates for anti-obesity treatment, although dedicated studies are lacking. This evidence gap may contribute to therapeutic inertia and limit equitable access to therapeutic innovation. We highlight the need for dedicated studies, real-world evidence, and expert recommendations to ensure that rare diseases, already orphan conditions by definition, do not become orphan indications in the era of modern obesity pharmacotherapy.