Virtual Town Hall - Gene Therapy for ccALD

With updates from bluebird bio

The FDA approved bluebird bio’s gene therapy on September 16, 2022. Gene therapy is a treatment option for some children with cerebral ALD that offers an additional option to allogenic hematopoietic stem cell transplant.

bluebird bio's gene therapy treatment is now commercially available at designated treatment centers in the United States.  The first patient to receive the treatment since FDA approval received the therapy in March of this year.  Please join us for this virtual town hall to hear from clinicians about their experience with gene therapy, and from bluebird bio to learn where gene therapy is being offered, and the process for safety monitoring and data sharing following FDA approval.  Please submit your questions in advance to [email protected]

Zoom link will be in e-receipt that follows registration.

Please check back soon!


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