共 50 条
- [1] Intracerebroventricular cerliponase alfa for CLN2 disease: Clinical practice considerations from US clinicsMOLECULAR GENETICS AND METABOLISM, 2020, 129 (02) : S158 - S158Wang, Raymond Y.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Orange Cty, Orange, CA 92668 USA Childrens Hosp Orange Cty, Orange, CA 92668 USAde los Reyes, Emily论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Childrens Hosp Orange Cty, Orange, CA 92668 USALehwald, Lenora论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Childrens Hosp Orange Cty, Orange, CA 92668 USAAugustine, Erika论文数: 0 引用数: 0 h-index: 0机构: Univ Rochester, Med Ctr, Rochester, NY 14642 USA Childrens Hosp Orange Cty, Orange, CA 92668 USABerry-Kravis, Elizabeth论文数: 0 引用数: 0 h-index: 0机构: Rush Univ, Med Ctr, Chicago, IL 60612 USA Childrens Hosp Orange Cty, Orange, CA 92668 USAButler, Karen论文数: 0 引用数: 0 h-index: 0机构: Lebonheur Childrens Hosp & Med Ctr, Memphis, TN USA Childrens Hosp Orange Cty, Orange, CA 92668 USACormier, Natalie论文数: 0 引用数: 0 h-index: 0机构: Texas Childrens Hosp, Houston, TX 77030 USA Childrens Hosp Orange Cty, Orange, CA 92668 USADemarest, Scott论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Colorado, Denver, CO USA Childrens Hosp Orange Cty, Orange, CA 92668 USALu, Sam论文数: 0 引用数: 0 h-index: 0机构: UCSF Benioff Childrens Hosp Oakland, Oakland, CA USA Childrens Hosp Orange Cty, Orange, CA 92668 USAMadden, Jacqueline论文数: 0 引用数: 0 h-index: 0机构: UCSF Benioff Childrens Hosp Oakland, Oakland, CA USA Childrens Hosp Orange Cty, Orange, CA 92668 USAOlaya, Joffre论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Orange Cty, Orange, CA 92668 USA Childrens Hosp Orange Cty, Orange, CA 92668 USASee, Susan论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Orange Cty, Orange, CA 92668 USA Childrens Hosp Orange Cty, Orange, CA 92668 USAVierhile, Amy论文数: 0 引用数: 0 h-index: 0机构: Univ Rochester, Med Ctr, Rochester, NY 14642 USA Childrens Hosp Orange Cty, Orange, CA 92668 USAWheless, James论文数: 0 引用数: 0 h-index: 0机构: Lebonheur Childrens Hosp & Med Ctr, Memphis, TN USA Childrens Hosp Orange Cty, Orange, CA 92668 USA论文数: 引用数: h-index:机构:Cohen-Pfeffer, Jessica论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Childrens Hosp Orange Cty, Orange, CA 92668 USAChu, Dorna论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Childrens Hosp Orange Cty, Orange, CA 92668 USALeal-Pardinas, Fernanda论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Childrens Hosp Orange Cty, Orange, CA 92668 USA
- [2] Intracerebroventricular Cerliponase Alfa for Neuronal Ceroid Lipofuscinosis Type 2 Disease: Clinical Practice Considerations From US ClinicsPEDIATRIC NEUROLOGY, 2020, 110 : 64 - 70Reyes, Emily de los论文数: 0 引用数: 0 h-index: 0机构: Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USALehwald, Lenora论文数: 0 引用数: 0 h-index: 0机构: Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USA Peyton Manning Childrens Hosp, Dept Pediat Neurol, Evansville, IN USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USAAugustine, Erika F.论文数: 0 引用数: 0 h-index: 0机构: Univ Rochester, Med Ctr, Dept Neurol, Rochester, NY 14642 USA Univ Rochester, Med Ctr, Dept Pediat, Rochester, NY 14642 USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USABerry-Kravis, Elizabeth论文数: 0 引用数: 0 h-index: 0机构: Rush Univ, Med Ctr, Dept Pediat, Chicago, IL 60612 USA Rush Univ, Med Ctr, Dept Neurol Sci, Chicago, IL 60612 USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USAButler, Karen论文数: 0 引用数: 0 h-index: 0机构: Le Bonheur Childrens Hosp, Neurosci Inst, Memphis, TN USA Univ Tennessee, Ctr Hlth Sci, Div Pediat Neurol, Memphis, TN 38163 USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USACormier, Natalie论文数: 0 引用数: 0 h-index: 0机构: Texas Childrens Hosp, Dept Pediat Neurosurg, Houston, TX 77030 USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USADemarest, Scott论文数: 0 引用数: 0 h-index: 0机构: Univ Colorado, Sch Med, Dept Pediat, Childrens Hosp Colorado, Denver, CO USA Univ Colorado, Sch Med, Dept Neurol, Childrens Hosp Colorado, Denver, CO USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USALu, Sam论文数: 0 引用数: 0 h-index: 0机构: UCSF Benioff Childrens Hosp Oakland, Dept Gastroenterol, Oakland, CA USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USAMadden, Jacqueline论文数: 0 引用数: 0 h-index: 0机构: UCSF Benioff Childrens Hosp Oakland, Dept Gastroenterol, Oakland, CA USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USAOlaya, Joffre论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Orange Cty, Neurosci Unit, Orange, CA 92668 USA Univ Calif, Irvine Sch Med, Dept Neurosurg, Orange, CA USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USASee, Susan论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Orange Cty, Orange, CA 92668 USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USAVierhile, Amy论文数: 0 引用数: 0 h-index: 0机构: Univ Rochester, Med Ctr, Dept Neurol, Rochester, NY 14642 USA Univ Rochester, Med Ctr, Dept Pediat, Rochester, NY 14642 USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USAWheless, James W.论文数: 0 引用数: 0 h-index: 0机构: Le Bonheur Childrens Hosp, Neurosci Inst, Memphis, TN USA Univ Tennessee, Ctr Hlth Sci, Div Pediat Neurol, Memphis, TN 38163 USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USA论文数: 引用数: h-index:机构:Cohen-Pfeffer, Jessica论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USAChu, Dorna论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USALeal-Pardinas, Fernanda论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USAWang, Raymond Y.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif, Irvine Sch Med, Dept Pediat, Orange, CA USA Childrens Hosp Orange Cty, CHOC Childrens Specialists, Dept Metab Disorders, Orange, CA 92668 USA Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat Neurol, 700 Childrens Dr, Columbus, OH 43205 USA
- [3] Clinical Pharmacokinetics and Pharmacodynamics of Cerliponase Alfa, Enzyme Replacement Therapy for CLN2 Disease by Intracerebroventricular AdministrationCTS-CLINICAL AND TRANSLATIONAL SCIENCE, 2021, 14 (02): : 635 - 644Kim, Aryun论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA 94949 USA BioMarin Pharmaceut Inc, Novato, CA 94949 USAGrover, Anita论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA 94949 USA BioMarin Pharmaceut Inc, Novato, CA 94949 USAHammon, Kevin论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA 94949 USA BioMarin Pharmaceut Inc, Novato, CA 94949 USAde Hart, Greg论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA 94949 USA BioMarin Pharmaceut Inc, Novato, CA 94949 USASlasor, Peter论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA 94949 USA BioMarin Pharmaceut Inc, Novato, CA 94949 USACherukuri, Anu论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA 94949 USA BioMarin Pharmaceut Inc, Novato, CA 94949 USAAjayi, Temitayo论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA 94949 USA BioMarin Pharmaceut Inc, Novato, CA 94949 USAJacoby, David论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA 94949 USA BioMarin Pharmaceut Inc, Novato, CA 94949 USASchulz, Angela论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany BioMarin Pharmaceut Inc, Novato, CA 94949 USASpecchio, Nicola论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu Childrens Hosp IRCCS, Rome, Italy BioMarin Pharmaceut Inc, Novato, CA 94949 USAde Los Reyes, Emily论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Ohio State Univ, Columbus, OH 43210 USA BioMarin Pharmaceut Inc, Novato, CA 94949 USAGissen, Paul论文数: 0 引用数: 0 h-index: 0机构: UCL, NIHR Great Ormond St Hosp, Biomed Res Ctr, London, England BioMarin Pharmaceut Inc, Novato, CA 94949 USAHenshaw, Joshua W.论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA 94949 USA BioMarin Pharmaceut Inc, Novato, CA 94949 USA
- [4] Cerliponase alfa for CLN2 disease, a promising therapyEXPERT OPINION ON ORPHAN DRUGS, 2020, 8 (11): : 445 - 454Aylward, Shawn C.论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Dept Pediat & Neurol, Columbus, OH 43205 USA Nationwide Childrens Hosp, Dept Pediat & Neurol, Columbus, OH 43205 USAPindrik, Jonathan论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Div Pediat Neurosurg, Columbus, OH USA Ohio State Univ, Dept Neurol Surg, Columbus, OH 43210 USA Nationwide Childrens Hosp, Dept Pediat & Neurol, Columbus, OH 43205 USAAbreu, Nicolas J.论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Dept Pediat & Neurol, Columbus, OH 43205 USA Nationwide Childrens Hosp, Dept Pediat & Neurol, Columbus, OH 43205 USACherny, W. Bruce论文数: 0 引用数: 0 h-index: 0机构: St Lukes Childrens Hosp, Dept Pediat Neurosurg, Boise, ID USA Nationwide Childrens Hosp, Dept Pediat & Neurol, Columbus, OH 43205 USAO'Neal, Matthew论文数: 0 引用数: 0 h-index: 0机构: Marshall Univ, Joan C Edwards Sch Med, Huntington, WV USA Nationwide Childrens Hosp, Dept Pediat & Neurol, Columbus, OH 43205 USAde Los Reyes, Emily论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Dept Pediat & Neurol, Columbus, OH 43205 USA Nationwide Childrens Hosp, Dept Pediat & Neurol, Columbus, OH 43205 USA
- [5] Study of Intraventricular Cerliponase Alfa for CLN2 DiseaseNEW ENGLAND JOURNAL OF MEDICINE, 2018, 378 (20): : 1898 - 1907Schulz, Angela论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Martinistr 52, D-20246 Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Martinistr 52, D-20246 Hamburg, GermanyAjayi, Temitayo论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut, Novato, CA USA Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Martinistr 52, D-20246 Hamburg, GermanySpecchio, Nicola论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu Pediat Hosp, IRCCS, Dept Neurosci, Rome, Italy Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Martinistr 52, D-20246 Hamburg, Germanyde Los Reyes, Emily论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Ohio State Univ, Columbus, OH 43210 USA Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Martinistr 52, D-20246 Hamburg, GermanyGissen, Paul论文数: 0 引用数: 0 h-index: 0机构: UCL Great Ormond St Inst Child Hlth, London, England Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Martinistr 52, D-20246 Hamburg, GermanyBallon, Douglas论文数: 0 引用数: 0 h-index: 0机构: Weill Cornell Med Coll, Citigrp Biomed Imaging Ctr, Dept Radiol, New York, NY USA Weill Cornell Med Coll, Citigrp Biomed Imaging Ctr, Dept Med Genet, New York, NY USA Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Martinistr 52, D-20246 Hamburg, GermanyDyke, Jonathan P.论文数: 0 引用数: 0 h-index: 0机构: Weill Cornell Med Coll, Citigrp Biomed Imaging Ctr, Dept Radiol, New York, NY USA Weill Cornell Med Coll, Citigrp Biomed Imaging Ctr, Dept Med Genet, New York, NY USA Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Martinistr 52, D-20246 Hamburg, GermanyCahan, Heather论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut, Novato, CA USA Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Martinistr 52, D-20246 Hamburg, GermanySlasor, Peter论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut, Novato, CA USA Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Martinistr 52, D-20246 Hamburg, GermanyJacoby, David论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut, Novato, CA USA Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Martinistr 52, D-20246 Hamburg, GermanyKohlschutter, Alfried论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Martinistr 52, D-20246 Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Martinistr 52, D-20246 Hamburg, Germany
- [6] Dose selection for intracerebroventricular cerliponase alfa in children with CLN2 disease, translation from animal to human in a rare genetic diseaseCTS-CLINICAL AND TRANSLATIONAL SCIENCE, 2021, 14 (05): : 1810 - 1821Hammon, Kevin论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USA BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USAde Hart, Greg论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USA BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USAVuillemenot, Brian R.论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USA BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USAKennedy, Derek论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USA BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USAMusson, Don论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USA BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USAO'Neill, Charles A.论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USA BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USAKatz, Martin L.论文数: 0 引用数: 0 h-index: 0机构: Univ Missouri, Sch Med, Mason Eye Inst, Columbia, MO USA Univ Missouri, Dept Bioengn, Columbia, MO USA BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USAHenshaw, Joshua W.论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USA BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USA
- [7] Immunogenicity to cerliponase alfa intracerebroventricular enzyme replacement therapy for CLN2 disease: Results from a Phase 1/2 studyCLINICAL IMMUNOLOGY, 2018, 197 : 68 - 76Cherukuri, Anu论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USA BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USACahan, Heather论文数: 0 引用数: 0 h-index: 0机构: CytomX Therapeut, Early Clin Dev & Safety, San Francisco, CA USA BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USAde Hart, Greg论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USA BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USAVan Tuyl, Andrea论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USA BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USASlasor, Peter论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Global Clin Sci, Novato, CA USA BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USABray, Laurie论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Global Clin Sci, Novato, CA USA BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USAHenshaw, Joshua论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USA BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USAAjayi, Temitayo论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Global Clin Sci, Novato, CA USA BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USAJacoby, Dave论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Global Clin Sci, Novato, CA USA BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USAO'Neill, Charles A.论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Pharmacol Sci, Novato, CA USA BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USASchweighardt, Becky论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USA BioMarin Pharmaceut Inc, Translat Sci, Novato, CA USA
- [8] Psychometric Validation of the CLN2 Quality of Life Questionnaire in Participants with CLN2 Disease Treated with Cerliponase AlfaHEALTHCARE, 2024, 12 (22)Due, Christina论文数: 0 引用数: 0 h-index: 0机构: BioMarin Int Ltd, London WC1A 2SL, England BioMarin Int Ltd, London WC1A 2SL, EnglandQuinn, Jennifer论文数: 0 引用数: 0 h-index: 0机构: Debiopharm Inc, CH-1006 Lausanne, Switzerland BioMarin Int Ltd, London WC1A 2SL, EnglandGissen, Paul论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Hosp Biomed Res Ctr, Natl Inst Hlth Res, London WC1N 1EH, England BioMarin Int Ltd, London WC1A 2SL, EnglandSchulz, Angela论文数: 0 引用数: 0 h-index: 0机构: Univ Hosp Hamburg Eppendorf, Dept Pediat, D-20246 Hamburg, Germany German Ctr Child & Adolescent Hlth DZKJ, Partner Site Hamburg, D-20251 Hamburg, Germany BioMarin Int Ltd, London WC1A 2SL, EnglandSpecchio, Nicola论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu Pediat Hosp, European Reference Network EpiCARE, IRCCS, Neurol Epilepsy & Movement Disorders Unit, I-00165 Rome, Italy Univ Hosp KU Leuven, B-3000 Leuven, Belgium BioMarin Int Ltd, London WC1A 2SL, Englandde los Reyes, Emily论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH 43205 USA BioMarin Int Ltd, London WC1A 2SL, EnglandButt, Thomas论文数: 0 引用数: 0 h-index: 0机构: BioMarin Int Ltd, London WC1A 2SL, England BioMarin Int Ltd, London WC1A 2SL, England
- [9] Real-world clinical outcomes of patients with CLN2 disease treated with cerliponase alfaFRONTIERS IN NEUROLOGY, 2025, 16Schulz, Angela论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, Germany German Ctr Child & Adolescent Hlth DZKJ Hamburg Pa, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, GermanySchwering, Christoph论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, GermanyWibbeler, Eva论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, GermanyWestermann, Lena Marie论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, GermanyHagenah, Luca论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, GermanyLezius, Susanne论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Inst Med Biometry & Epidemiol, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, GermanyJha, Ashok论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, GermanyHunt, Abigail论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, GermanySlasor, Peter论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, GermanyReisewitz, Pascal论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, GermanyNickel, Miriam论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, Germany
- [10] Cerliponase Alfa for the Treatment of Atypical Phenotypes of CLN2 Disease: A Retrospective Case SeriesJOURNAL OF CHILD NEUROLOGY, 2021, 36 (06) : 468 - 474Wibbeler, Eva论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, GermanyWang, Raymond论文数: 0 引用数: 0 h-index: 0机构: CHOC Childrens Specialists, Orange, CA USA Univ Calif Irvine, Sch Med, Irvine, CA 92717 USA Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, Germanyde los Reyes, Emily论文数: 0 引用数: 0 h-index: 0机构: Ohio State Univ, Nationwide Children Hosp Columbus Ohio, Columbus, OH 43210 USA Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, GermanySpecchio, Nicola论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu, Rome, Italy Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, GermanyGissen, Paul论文数: 0 引用数: 0 h-index: 0机构: NIHR Great Ormond St Hosp, Biomed Res Ctr, London, England Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, GermanyGuelbert, Norberto论文数: 0 引用数: 0 h-index: 0机构: Holy Trinity Childrens Hosp, Cordoba, Argentina Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, GermanyNickel, Miriam论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, GermanySchwering, Christoph论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, GermanyLehwald, Lenora论文数: 0 引用数: 0 h-index: 0机构: Ohio State Univ, Nationwide Children Hosp Columbus Ohio, Columbus, OH 43210 USA Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, GermanyTrivisano, Marina论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu, Rome, Italy Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, GermanyLee, Laura论文数: 0 引用数: 0 h-index: 0机构: NIHR Great Ormond St Hosp, Biomed Res Ctr, London, England Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, GermanyAmato, Gianni论文数: 0 引用数: 0 h-index: 0机构: Biostats LLC, Las Vegas, NV USA Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, GermanyCohen-Pfeffer, Jessica论文数: 0 引用数: 0 h-index: 0机构: BioMarin, Novato, CA USA Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, GermanyShediac, Renee论文数: 0 引用数: 0 h-index: 0机构: BioMarin, Novato, CA USA Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, GermanyLeal-Pardinas, Fernanda论文数: 0 引用数: 0 h-index: 0机构: BioMarin, Novato, CA USA Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, GermanySchulz, Angela论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Childrens Hosp, Hamburg, Germany