Proper Name: elivaldogene autotemcel
Manufacturer: bluebird bio, Inc.
- To slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD).
- Package Insert - SKYSONA
- Demographic Subgroup Information – elivaldogene autotemcel [SKYSONA]
Refer to Section 1.1 of the Clinical Review Memo for information about participation in the clinical trials and any analysis of demographic subgroup outcomes that is notable.