Virtual Town Hall: Gene Therapy Treatment for childhood cerebral Adrenoleukodystrophy (ALD)
Автор: ALD Alliance
Загружено: Дата премьеры: 20 янв. 2023 г.
Просмотров: 987 просмотров
The FDA approved bluebird bio’s gene therapy treatment for childhood cerebral adrenoleukodystrophy (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. Bluebird bio made their gene therapy treatment commercially available in the beginning of 2023 at designated treatment centers. Dr. Christy Duncan, pediatric stem cell transplant physician, provides a medical overview of the treatment process. Representatives from bluebird bio follow with information about their supportive services and treatment locations. The program concludes with a brief Q&A.
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