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Targeted Drug Shows Promise in Advanced Form of Mastocytosis

In a Phase 1 trial, patients with an advanced or aggressive form of systemic mastocytosis had rapid and durable responses with few adverse effects following treatment with an investigational drug, BLU-285 that targets the genetic mutation called KIT D816V found in more than 90% of the cases. The study was presented at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta.