Bluebird Bio’s Skysona Receives FDA Approval and Becomes World’s Most Expensive Drug
XTalks
SEPTEMBER 27, 2022
The record-breaking price tag of Skysona is not surprising for one-time gene therapies like it. CALD is caused by mutations in the ABCD1 gene that affect the production of adrenoleukodystrophy protein (ALDP), which leads to the accumulation of very long-chain fatty acids (VLCFAs), mainly in the white matter of the brain and spinal cord.
52 
Let's personalize your content