共 50 条
- [1] AAV mediated gene therapy to treat loss of vision in CLN2 (Batten disease)HUMAN GENE THERAPY, 2017, 28 (12) : A82 - A82Aristorena, M.论文数: 0 引用数: 0 h-index: 0机构: UCL Inst Ophthalmol, London, England UCL Inst Ophthalmol, London, EnglandHolthaus, S. M. Kleine论文数: 0 引用数: 0 h-index: 0机构: UCL Inst Ophthalmol, London, England UCL Inst Ophthalmol, London, EnglandPearson, R. A.论文数: 0 引用数: 0 h-index: 0机构: UCL Inst Ophthalmol, London, England UCL Inst Ophthalmol, London, EnglandRahim, A. A.论文数: 0 引用数: 0 h-index: 0机构: UCL Sch Pharm, London, England UCL Inst Ophthalmol, London, EnglandSmith, A. J.论文数: 0 引用数: 0 h-index: 0机构: UCL Inst Ophthalmol, London, England UCL Inst Ophthalmol, London, EnglandMole, S. E.论文数: 0 引用数: 0 h-index: 0机构: UCL MRC Lab Mol Cell Biol, London, England UCL Inst Ophthalmol, London, EnglandAli, R. R.论文数: 0 引用数: 0 h-index: 0机构: UCL Inst Ophthalmol, London, England UCL Inst Ophthalmol, London, England
- [2] 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
- [3] Management Strategies for CLN2 DiseasePEDIATRIC NEUROLOGY, 2017, 69 : 102 - 112Williams, Ruth E.论文数: 0 引用数: 0 h-index: 0机构: Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, England Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandAdams, Heather R.论文数: 0 引用数: 0 h-index: 0机构: Univ Rochester, Dept Neurol, Sch Med, Rochester, NY USA Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandBlohm, Martin论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, Germany Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandCohen-Pfeffer, Jessica L.论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, Englandde los Reyes, Emily论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Dept Pediat Neurol, Columbus, OH USA Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandDenecke, Jonas论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, Germany Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandDrago, Kristen论文数: 0 引用数: 0 h-index: 0机构: Journeycare Children, Chicago, IL USA Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandFairhurst, Charlie论文数: 0 引用数: 0 h-index: 0机构: Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, England Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandFrazier, Margie论文数: 0 引用数: 0 h-index: 0机构: BDSRA, Columbus, OH USA Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandGuelbert, Norberto论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Cordoba, Metab Dis Sect, Cordoba, Argentina Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandKiss, Szilard论文数: 0 引用数: 0 h-index: 0机构: Weill Cornell Med Coll, Dept Ophthalmol, New York, NY USA Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandKofler, Annamaria论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu Pediat Hosp, IRCCS, Dept Neurosci, Rome, Italy Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandLawson, John A.论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp, Dept Neurol, Randwick, NSW, Australia Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandLehwald, Lenora论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Dept Pediat Neurol, Columbus, OH USA Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandLeung, Mary-Anne论文数: 0 引用数: 0 h-index: 0机构: Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, England Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandMikhaylova, Svetlana论文数: 0 引用数: 0 h-index: 0机构: Russian Childrens Clin Hosp, Dept Med Genet, Moscow, Russia Russian Natl Res Med Univ, Dept Mol & Cell Genet, Moscow, Russia Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandMink, Jonathan W.论文数: 0 引用数: 0 h-index: 0机构: Univ Rochester, Dept Neurol, Sch Med, Rochester, NY USA Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandNickel, Miriam论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, Germany Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandShediac, Renee论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandSims, Katherine论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Dept Neurol, Boston, MA 02114 USA Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandSpecchio, Nicola论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu Pediat Hosp, IRCCS, Dept Neurosci, Rome, Italy Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandTopcu, Meral论文数: 0 引用数: 0 h-index: 0机构: Hacettepe Univ, Dept Pediat Neurol, Ankara, Turkey Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, Englandvon Loebbecke, Ina论文数: 0 引用数: 0 h-index: 0机构: Practice Paediat Physiotherapy, Hamburg, Germany Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, EnglandWest, Andrea论文数: 0 引用数: 0 h-index: 0机构: BDFA, Farnborough, Hants, England Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, England论文数: 引用数: h-index:机构:Schulz, Angela论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Hamburg, Germany Evelina London Childrens Hosp, Childrens Neurosci Ctr, London, England
- [4] Changing Times for CLN2 Disease: The Era of Enzyme Replacement TherapyTHERAPEUTICS AND CLINICAL RISK MANAGEMENT, 2020, 16 : 213 - 222Specchio, Nicola论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu Pediat Hosp, IRCCS, Dept Neurosci, Rare & Complex Epilepsy Unit, Rome, Italy Bambino Gesu Pediat Hosp, IRCCS, Dept Neurosci, Rare & Complex Epilepsy Unit, Rome, ItalyPietrafusa, Nicola论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu Pediat Hosp, IRCCS, Dept Neurosci, Rare & Complex Epilepsy Unit, Rome, Italy Bambino Gesu Pediat Hosp, IRCCS, Dept Neurosci, Rare & Complex Epilepsy Unit, Rome, ItalyTrivisano, Marina论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu Pediat Hosp, IRCCS, Dept Neurosci, Rare & Complex Epilepsy Unit, Rome, Italy Bambino Gesu Pediat Hosp, IRCCS, Dept Neurosci, Rare & Complex Epilepsy Unit, Rome, Italy
- [5] Monitoring Neuropsychological Function in CLN2 DiseaseANNALS OF NEUROLOGY, 2018, 84 : S401 - S402Augustine, E.论文数: 0 引用数: 0 h-index: 0Adams, H.论文数: 0 引用数: 0 h-index: 0Baron, I. S.论文数: 0 引用数: 0 h-index: 0Bjoraker, K.论文数: 0 引用数: 0 h-index: 0Cohen-Pfeffer, J.论文数: 0 引用数: 0 h-index: 0Delaney, K.论文数: 0 引用数: 0 h-index: 0Elmerskog, B.论文数: 0 引用数: 0 h-index: 0Newsom-Davis, I论文数: 0 引用数: 0 h-index: 0Nickel, G.论文数: 0 引用数: 0 h-index: 0Rust, S.论文数: 0 引用数: 0 h-index: 0Shapiro, E.论文数: 0 引用数: 0 h-index: 0Tossebro, A. G.论文数: 0 引用数: 0 h-index: 0Schulz, A.论文数: 0 引用数: 0 h-index: 0
- [6] Expert opinion on the management of CLN2 diseaseMOLECULAR GENETICS AND METABOLISM, 2016, 117 (02) : S103 - S104Schulz, Angela论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyAdams, Heather R.论文数: 0 引用数: 0 h-index: 0机构: Univ Rochester, Sch Med, Rochester, NY USA Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyBlohm, Martin论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyCohen-Pfeffer, Jessica L.论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Univ Med Ctr Hamburg Eppendorf, Hamburg, Germanyde los Reyes, Emily论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyDenecke, Jonas论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyDrago, Kristen论文数: 0 引用数: 0 h-index: 0机构: Joumeycare Children, Chicago, IL USA Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyFairhurst, Charlie论文数: 0 引用数: 0 h-index: 0机构: Evelina London Childrens Hosp, London, England Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyFrazier, Margie论文数: 0 引用数: 0 h-index: 0机构: BDSRA, Columbus, OH USA Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyGuelbert, Norberto论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Cordoba, Cordoba, Argentina Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyKiss, Szilard论文数: 0 引用数: 0 h-index: 0机构: Weill Cornell Med Coll, New York, NY USA Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyKofler, Annamaria论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu Pediat Hosp, IRCCS, Rome, Italy Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyLawson, John论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp, Sydney, NSW, Australia Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyLehwald, Lenora论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyLeung, Mary-Anne论文数: 0 引用数: 0 h-index: 0机构: Evelina London Childrens Hosp, London, England Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyMikhailova, Svetlana论文数: 0 引用数: 0 h-index: 0机构: Russian Reg Pediat Hosp, Moscow, Russia Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyMink, Jonathan W.论文数: 0 引用数: 0 h-index: 0机构: Univ Rochester, Sch Med, Rochester, NY USA Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyNickel, Miriam论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyShediac, Renee论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanySims, Katherine论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Boston, MA 02114 USA Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanySpecchio, Nicola论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu Pediat Hosp, IRCCS, Rome, Italy Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyTopcu, Meral论文数: 0 引用数: 0 h-index: 0机构: Hacettepe Univ, Ankara, Turkey Univ Med Ctr Hamburg Eppendorf, Hamburg, Germanyvon Loebbecke, Ina论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyWest, Andrea论文数: 0 引用数: 0 h-index: 0机构: BDFA, Farnborough, Hants, England Univ Med Ctr Hamburg Eppendorf, Hamburg, GermanyWilliams, Ruth E.论文数: 0 引用数: 0 h-index: 0机构: Evelina London Childrens Hosp, London, England Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany
- [7] Retinal degeneration in patients with CLN2 diseaseINVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2019, 60 (09)Dulz, Simon论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Ophthalmol, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Ophthalmol, Hamburg, GermanySchwering, Christoph论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp, Med Univ Ctr Hamburg Eppendorf, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Ophthalmol, Hamburg, GermanyWibbeler, Eva论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp, Med Univ Ctr Hamburg Eppendorf, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Ophthalmol, Hamburg, GermanyNickel, Miriam论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp, Med Univ Ctr Hamburg Eppendorf, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Ophthalmol, Hamburg, GermanySpitzer, Martin论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Ophthalmol, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Ophthalmol, Hamburg, GermanySchulz, Angela论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp, Med Univ Ctr Hamburg Eppendorf, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Ophthalmol, Hamburg, GermanyAtiskova, Yevgeniya论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Ophthalmol, Hamburg, Germany Univ Med Ctr Hamburg Eppendorf, Ophthalmol, Hamburg, Germany
- [8] EXPERT OPINION ON THE MANAGEMENT OF CLN2 DISEASEMOLECULAR GENETICS AND METABOLISM, 2016, 117 (03) : 293 - 293Williams, Ruth E.论文数: 0 引用数: 0 h-index: 0机构: Evelina London Childrens Hosp, London, England Evelina London Childrens Hosp, London, EnglandCohen-Pfeffer, Jessica论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Evelina London Childrens Hosp, London, EnglandAdams, Heather论文数: 0 引用数: 0 h-index: 0机构: Univ Rochester, Sch Med, Rochester, NY USA Evelina London Childrens Hosp, London, EnglandBlohm, Martin论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany Evelina London Childrens Hosp, London, Englandde los Reyes, Emily论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Evelina London Childrens Hosp, London, EnglandDenecke, Jonas论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany Evelina London Childrens Hosp, London, EnglandDrago, Kristen论文数: 0 引用数: 0 h-index: 0机构: Journeycare Children, Chicago, IL USA Evelina London Childrens Hosp, London, EnglandFairhurst, Charlie论文数: 0 引用数: 0 h-index: 0机构: Evelina London Childrens Hosp, London, England Evelina London Childrens Hosp, London, EnglandFrazier, Margie论文数: 0 引用数: 0 h-index: 0机构: BDSRA, Columbus, OH USA Evelina London Childrens Hosp, London, EnglandGuelbert, Norberto论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Cordoba, Cordoba, Argentina Evelina London Childrens Hosp, London, EnglandKiss, Szilard论文数: 0 引用数: 0 h-index: 0机构: Weill Cornell Med Coll, New York, NY USA Evelina London Childrens Hosp, London, EnglandKofler, Annamaria论文数: 0 引用数: 0 h-index: 0机构: IRCCS, Bambino Gesu Childrens Hosp, Rome, Italy Evelina London Childrens Hosp, London, EnglandLawson, John论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp, Sydney, NSW, Australia Evelina London Childrens Hosp, London, EnglandLehwald, Lenora论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Evelina London Childrens Hosp, London, EnglandLeung, Mary-Anne论文数: 0 引用数: 0 h-index: 0机构: Evelina London Childrens Hosp, London, England Evelina London Childrens Hosp, London, EnglandMikhailova, Svetlana论文数: 0 引用数: 0 h-index: 0机构: Russian Reg Pediat Hosp, Moscow, Russia Evelina London Childrens Hosp, London, EnglandMink, Jonathan论文数: 0 引用数: 0 h-index: 0机构: Univ Rochester, Sch Med, Rochester, NY USA Evelina London Childrens Hosp, London, EnglandNickel, Miriam论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany Evelina London Childrens Hosp, London, EnglandShediac, Renee论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA Evelina London Childrens Hosp, London, EnglandSims, Katherine论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Boston, MA 02114 USA Evelina London Childrens Hosp, London, EnglandSpecchio, Nicola论文数: 0 引用数: 0 h-index: 0机构: IRCCS, Bambino Gesu Childrens Hosp, Rome, Italy Evelina London Childrens Hosp, London, EnglandTopcu, Meral论文数: 0 引用数: 0 h-index: 0机构: Hacettepe Univ, Ankara, Turkey Evelina London Childrens Hosp, London, Englandvon Lobbecke, Ina论文数: 0 引用数: 0 h-index: 0机构: Evelina London Childrens Hosp, London, EnglandWest, Andrea论文数: 0 引用数: 0 h-index: 0机构: BDFA, Farnborough, Hants, England Evelina London Childrens Hosp, London, EnglandSchulz, Angela论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany Evelina London Childrens Hosp, London, England
- [9] OCT Biomarkers in Ocular CLN2 DiseaseINVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2021, 62 (08)Huang, Wei Chieh论文数: 0 引用数: 0 h-index: 0机构: REGENXBIO Inc, Rockville, MD USA REGENXBIO Inc, Rockville, MD USAAtiskova, Yevgeniya论文数: 0 引用数: 0 h-index: 0机构: Univ Klinikum Hamburg Eppendorf, Dept Ophthalmol, Hamburg, Germany REGENXBIO Inc, Rockville, MD USAOhnsman, Christina M.论文数: 0 引用数: 0 h-index: 0机构: REGENXBIO Inc, Rockville, MD USA REGENXBIO Inc, Rockville, MD USADulz, Simon论文数: 0 引用数: 0 h-index: 0机构: Univ Klinikum Hamburg Eppendorf, Dept Ophthalmol, Hamburg, Germany REGENXBIO Inc, Rockville, MD USA
- [10] 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