X-linked adrenoleukodystrophy (X-ALD) is a debilitating disease caused by mutations in the ABCD1 gene. Approximately 30% of affected boys develop the progressive ...
Skysona is a one-time gene therapy designed to add functional copies of the ABCD1 cDNA into a patient’s own hematopoietic stem cells, resulting in the production of functional ALDP. Treatment with ...
Credit: Getty Images. Elivaldogene autotemcel (eli-cel) is a one-time gene therapy designed to add functional copies of the ABCD1 gene into a patient’s own ...
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