A Case Series on Genotype and Outcome of Liver Transplantation in Children with Niemann-Pick Disease Type C

• Main Authors: Line Modin (Author), Vicky Ng (Author), Paul Gissen (Author), Julian Raiman (Author), Eva Doreen Pfister (Author), Anibh Das (Author), René Santer (Author), Hanna Faghfoury (Author), Saikat Santra (Author), Ulrich Baumann (Author)
• Format: Book
• Published: MDPI AG, 2021-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Background: To report on clinical presentation and outcomes of children who underwent liver transplantation (LTx) and were subsequently diagnosed to have Niemann-Pick type C (NPC). Methods: Retrospective, descriptive, multi-centre review of children diagnosed with NPC who underwent LTx (2003-2018)....