bluebird的gene therapy被调查了,可能导致血癌

h
hululy
楼主 (北美华人网)
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.
H
Huarenxuan
这是治疗什么病的?
m
minglan
回复 2楼 Huarenxuan 的帖子
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