As drugmakers take lessons from oncology in developing CAR T cells for other diseases, they must contend with risks that ...
The bill would establish the right of cancer patients to access advanced care, including precision medicine and genomic testing, in their communities.
The agency had previously recommended against marketing authorization for the Alzheimer's disease but reconsidered after a review.
The panels assess genes with strong disease links and clinical management guidelines, but the firm offers limited-evidence genes as an add-on option.
This is the first gene therapy for AADC deficiency to receive US regulatory approval and the first that can be administered directly to the brain.