Indications
Treatment of early cerebral adrenoleukodystrophy in patients less than 18 years of age, with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA) matched sibling haematopoietic stem cell donor is not available.
Composition
- Active substance
- elivaldogene autotemcel
Official product information
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At a glance
- Medicine
- Skysona
- Active substance
- elivaldogene autotemcel
- ATC code
- N07
- Group
- Nervous system
- Therapeutic area
- Adrenoleukodystrophy
- Marketing authorisation holder
- bluebird bio (Netherlands) B.V.
- Status
- Withdrawn