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NICE recommends Koselugo for neurofibromatosis type 1

 Last Update: 2022-05-18
 Source: Internet
 Author: User

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The National Institute for Health and Care Excellence (NICE) has recommended AstraZeneca and MSD's Koselugo (selumetinib) for use in the UK NHS

child

NF1 is a debilitating genetic disorder with a prevalence of 1 in 4,000 people

Koselugo works by blocking specific enzymes -- mitogen-activated protein kinase kinases 1 and 2 (also known as MEK 1 and MEK2) that are involved in stimulating cell growth

An open-label phase II trial determined the clinical benefit of selumetinib in patients with plexiform neurofibromas

As of March 29, 2019, a total of 34 patients (68%) had confirmed partial responses, of which 28 had durable responses (lasting ≥1 year)

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