Thank you to everyone who joined us in person and virtually for the 8th Annual ALD Standards of Care Conference in March 2024. The presentation videos are now available on our YouTube channel.

Thank you to our 2024 ALD Standards of Care Conference Sponsors:

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Virtual Town Hall: Gene Therapy for childhood cALD

​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 2023.  The ALD Alliance hosted this second virtual town hall in July 2023 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.  
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