FDA approved first therapy for children with debilitating and disfiguring rare disease

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On Apr. 10, 2020, the FDA approved AstraZeneca ‘s Koselugo (selumetinib) for treatment of pediatric patients, 2 years of age and older, with neurofibromatosis type 1 (NF1), a genetic disorder of the nervous system causing tumors to grow on nerves. Koselugo is the first drug approved by the FDA to treat this debilitating, progressive and often disfiguring rare disease that typically begins early in life.

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Source: U.S. Food and Drug Administration
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