FDA probes blood cancer risk from bluebird's gene therapy, weighs regulatory action Nov 27 (Reuters) - The U.S. Food and Drug Administration is weighing the need for regulatory action on bluebird bio's (BLUE.O), opens new tab gene therapy for a rare neurological disorder, it said on Wednesday, as the agency probes additional reports of blood cancers from its use. Bluebird's Skysona was approved by the FDA in 2022 for the treatment of cerebral adrenoleukodystrophy (CALD) and was priced at $3 million at the time of its launch. CALD, which affects about 1 in 20,000 to 50,000 people globally, typically occurs in boys aged between 3 and 12 years.
Nov 27 (Reuters) - The U.S. Food and Drug Administration is weighing the need for regulatory action on bluebird bio's (BLUE.O), opens new tab gene therapy for a rare neurological disorder, it said on Wednesday, as the agency probes additional reports of blood cancers from its use. Bluebird's Skysona was approved by the FDA in 2022 for the treatment of cerebral adrenoleukodystrophy (CALD) and was priced at $3 million at the time of its launch. CALD, which affects about 1 in 20,000 to 50,000 people globally, typically occurs in boys aged between 3 and 12 years.
一楼写了简称cald，什么dystrophy，貌似某种罕见神经系统的退行性病变