mTOR inhibitors reduce enteropathy, intestinal bleeding and colectomy rate in patients with juvenile polyposis of infancy with PTEN-BMPR1A deletion.

Hum Mol Genet · 2021 · PMC8804886 · PMID 33822054

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highly significantno p-value reported

However, the treatment effect observed is large, highly significant and transformative for patients.

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