First-ever lentiviral hematopoietic stem cell gene therapy approved for reimbursement by NHS England Two children with early-onset MLD recently treated commercially with Libmeldy in Germany and France A third eligible ...
Administration of one-time hematopoietic stem cell (HSC) gene therapy resulted in sustained, clinically meaningful benefits by preserving cognitive function and motor development in most patients 90% overall survival with up ...
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