Gene Therapy & Adrenoleukodystrophy (ALD)

The FDA approved bluebird bio’s gene therapy treatment for childhood cerebral ALD on September 16, 2022. Gene therapy is a treatment option for children with cerebral ALD that offers an alternative to allogenic hematopoietic stem cell transplant, eliminating the need for a bone marrow match. SKYSONA® (elivaldogene autotemcel), also known as eli-cel, is now commercially available at designated treatment centers in the United States. Bluebird bio’s patient services program, my bluebird support, which is appropriate for both caregivers and clinicians to use, can be reached at: 1-833-888-NEST (6378) Monday–Friday, 8 am–8 pm ET or online at

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