Transplantation as disease modifying therapy in the era of gene therapy medicinal products - health policy considerations.
Wagenmakers M, Lehman A, Hoed CD, Dussen LV, Langeveld M, Sirrs S
Orphanet journal of rare diseases, 2026 Aug 13
Abstract
New gene therapy medicinal products [GTMP] are being considered as alternatives to liver transplant [LTx] for some patients with inherited metabolic diseases [IMDs] but pose unique challenges for health policy makers.