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Christian J. Hendriksz
Researcher at University of Pretoria
Publications - 135
Citations - 4904
Christian J. Hendriksz is an academic researcher from University of Pretoria. The author has contributed to research in topics: Elosulfase alfa & Morquio syndrome. The author has an hindex of 33, co-authored 135 publications receiving 4018 citations. Previous affiliations of Christian J. Hendriksz include University of Manchester & UCL Institute of Child Health.
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Journal ArticleDOI
Recommendations for the diagnosis and management of Niemann-Pick disease type C: an update.
Marc C. Patterson,Christian J. Hendriksz,Mark Walterfang,Frédéric Sedel,Marie T. Vanier,Frits A. Wijburg +5 more
TL;DR: Points of consensus among experts in the diagnosis and treatment of NP-C are reported based on a follow-up meeting in Paris, France in September 2011, providing further information on detection/diagnostic methods, potential new methods of monitoring disease progression, and therapy.
Journal ArticleDOI
Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document
Marieke Biegstraaten,Reynir Arngrímsson,Frédéric Barbey,Lut Boks,Franco Cecchi,Patrick Deegan,Ulla Feldt-Rasmussen,Tarekegn Geberhiwot,Dominique P. Germain,Christian J. Hendriksz,Derralynn Hughes,Ilkka Kantola,Nesrin Karabul,Christine Lavery,Gabor E. Linthorst,Atul Mehta,Erica van de Mheen,João Paulo Oliveira,Rossella Parini,Uma Ramaswami,Michael A. Rudnicki,Andreas L. Serra,Claudia Sommer,Gere Sunder-Plassmann,Einar Svarstad,Annelies Sweeb,Wim Terryn,Anna Tylki-Szymańska,Camilla Tøndel,Bojan Vujkovac,Frank Weidemann,Frits A. Wijburg,Peter Woolfson,Carla E. M. Hollak +33 more
TL;DR: In this article, the authors defined European consensus recommendations for the initiation and cessation of Enzyme Replacement Therapy (ERT) in patients with Fabry disease, which may halt or attenuate disease progression.
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Phenotypic spectrum of neurodegeneration associated with mutations in the PLA2G6 gene (PLAN)
M. A. Kurian,Neil V. Morgan,Lesley MacPherson,Keith Foster,D. Peake,R. Gupta,Sunny Philip,Christian J. Hendriksz,Jenny Morton,H. M. Kingston,Elisabeth Rosser,Evangeline Wassmer,Paul Gissen,Eamonn R. Maher +13 more
TL;DR: Although patients with PLAN have previously been diagnosed with infantile neuroaxonal dystrophy, neurodegeneration associated with brain iron accumulation, and Karak syndrome, they display a characteristic clinical and radiologic phenotype that will negate the need for more invasive diagnostic procedures such as tissue biopsy.
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Consensus clinical management guidelines for Niemann-Pick disease type C.
Tarekegn Geberhiwot,Alessandro Moro,Andrea Dardis,Uma Ramaswami,Sandra Sirrs,Mercedes Pineda Marfa,Marie T. Vanier,Mark Walterfang,Shaun Bolton,Charlotte Dawson,Bénédicte Héron,Miriam Stampfer,Jackie Imrie,Christian J. Hendriksz,Paul Gissen,Ellen Crushell,Maria Josep Coll,Yann Nadjar,Hans H. Klünemann,Eugen Mengel,M. Hrebicek,Simon Jones,Daniel S. Ory,Bruno Bembi,Marc C. Patterson +24 more
TL;DR: Clinical guidelines that define standard of care for NPC patients, foster shared care arrangements between expert centres and family physicians, and empower patients are developed that can inform care providers, care funders, patients and their carers of best practice of care.
Journal ArticleDOI
Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study
Christian J. Hendriksz,Christian J. Hendriksz,Barbara K. Burton,Thomas R. Fleming,Paul Harmatz,Derralynn Hughes,Simon Jones,Shuan-Pei Lin,Eugen Mengel,Maurizio Scarpa,Vassili Valayannopoulos,Roberto Giugliani,Strive Investigators,Peter Slasor,Debra L. Lounsbury,Wolfgang Dummer +15 more
TL;DR: Elosulfase alfa improved endurance as measured by the 6MWT in the weekly but not qow dose group, did not improve endurance on the 3MSCT, reduced urine KS, and had an acceptable safety profile.