Search Results - Paul Gissen

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  1. 1
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    Diagnostic and Management Issues in Patients with Late-Onset Ornithine Transcarbamylase Deficiency by Majitha Seyed Ibrahim, Jessica I. Gold, Alison Woodall, Berna Seker Yilmaz, Paul Gissen, Karolina M. Stepien

    Published 2023
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    A Case Series on Genotype and Outcome of Liver Transplantation in Children with Niemann-Pick Disease Type C by Line Modin, Vicky Ng, Paul Gissen, Julian Raiman, Eva Doreen Pfister, Anibh Das, René Santer, Hanna Faghfoury, Saikat Santra, Ulrich Baumann

    Published 2021
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    Clinical Pharmacokinetics and Pharmacodynamics of Cerliponase Alfa, Enzyme Replacement Therapy for CLN2 Disease by Intracerebroventricular Administration by Aryun Kim, Anita Grover, Kevin Hammon, Greg deHart, Peter Slasor, Anu Cherukuri, Temitayo Ajayi, David Jacoby, Angela Schulz, Nicola Specchio, Emily deLos Reyes, Paul Gissen, Joshua W. Henshaw

    Published 2021
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    Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy by Haiyan Zhou, Jinhong Meng, Alberto Malerba, Francesco Catapano, Palittiya Sintusek, Susan Jarmin, Lucy Feng, Ngoc Lu‐Nguyen, Lianwen Sun, Virginie Mariot, Julie Dumonceaux, Jennifer E. Morgan, Paul Gissen, George Dickson, Francesco Muntoni

    Published 2020
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