SKYSONA™ TREATS EARLY, ACTIVE CEREBRAL ALD
SKYSONA is a one-time gene therapy that slows the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD).*
*Early, active CALD refers to asymptomatic or mildly symptomatic (NFS ≤ 1) boys who have gadolinium enhancement on brain MRI and Loes scores of 0.5-9.
How does SKYSONA work?
SKYSONA is made specifically for each patient using the patient’s own blood stem cells, and adds functional copies of the ABCD1 gene to their cells.
This addition allows the child’s body to make ALD protein (ALDP), which helps their body to break down very long–chain fatty acids (VLCFAs) in the brain. This process slows the progression of damage to the brain and slows the decline in neurologic function.
Your child’s blood stem cells are collected
SKYSONA is made specifically by using your child’s own blood stem cells.
SKYSONA IS MANUFACTURED
Once a child’s blood stem cells have been collected, working copies of the ABCD1 gene are added to these cells through the aid of a viral vector.
SKYSONA CELLS REPRODUCE
After SKYSONA is administered, it makes its way to the child’s bone marrow, where it goes through engraftment (making other healthy blood cells).
FUNCTIONAL ALDP IS PRODUCED
Genetically modified cells allow the child’s body to make ALDP.
ALDP BREAKS DOWN VLCFAs IN THE BRAIN
With functional ALDP, the child is able to effectively break down VLCFAs from their brain—slowing the progression of damage to the brain and slowing the decline in neurologic function.
Connect with a Patient Navigator –
a specialist from my bluebird support who can help you navigate through the SKYSONA treatment process:
Connect with a
Patient Navigator –
a specialist from my bluebird support who can help you navigate through the SKYSONA treatment process: