Treating early, active cerebral adrenoleukodystrophy (CALD)

In two 24-month, open-label, single-arm studies in patients with early, active CALD, estimated MFD-free survival at Month 24 from time of symptom onset (first NFS ≥ 1) was 72% (95% CI: 35%, 90%) for the symptomatic SKYSONA® treated patients (n=11) and 43% (95% CI: 10%, 73%) for the untreated patients (n=7; Natural History Population).*†

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*Based on post hoc enrichment analysis in symptomatic patients that compared time from onset of symptoms (NFS ≥ 1) to time to first MFD or death (ie, MFD-free survival) in SKYSONA treated and untreated natural history controls whose data derived from existing medical records for patients with CALD.

†The MFDs are defined as: loss of communication, cortical blindness, requirement for tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement. To be included in the analysis, patients had to have symptoms at baseline (NFS=1) or be asymptomatic (NFS=0) at baseline and have developed symptoms (NFS ≥ 1) during the course of follow-up in the study. Additionally, they had to have at least 24 months of follow-up after initial NFS ≥ 1 or have had an event (MFD or death).

MFD = major functional disability; NFS = neurologic function score.

Treat early, active cerebral adrenoleukodystrophy (CALD) with SKYSONA Treat early, active cerebral adrenoleukodystrophy (CALD) with SKYSONA
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Treating early, active cerebral adrenoleukodystrophy (CALD)

Who is SKYSONA for?

SKYSONA is the first and only approved gene therapy that slows the progression of neurologic dysfunction in boys with early, active CALD.‡ SKYSONA is for patients who meet these criteria:
Early active CALD

Early,
active
CALD

4-17 years of age

4–17
years of
age

‡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.

MRI = magnetic resonance imaging.

Learn more about SKYSONA by exploring these resources

SKYSONA
Prescribing Information SKYSONA
Medication Guide SKYSONA
Caregiver Brochure
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  • agree for bluebird bio, Inc. and companies working on behalf of bluebird bio, Inc., to use the information provided on this form to stay in contact with you for marketing and educational purposes and/or provide information about products and activities from bluebird bio, Inc.
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