{"id":"https://openalex.org/W3023933160","doi":"https://doi.org/10.1016/j.pediatrneurol.2020.04.018","title":"Intracerebroventricular Cerliponase Alfa for Neuronal Ceroid Lipofuscinosis Type 2 Disease: Clinical Practice Considerations From US Clinics","display_name":"Intracerebroventricular Cerliponase Alfa for Neuronal Ceroid Lipofuscinosis Type 2 Disease: Clinical Practice Considerations From US Clinics","publication_year":2020,"publication_date":"2020-05-04","ids":{"openalex":"https://openalex.org/W3023933160","doi":"https://doi.org/10.1016/j.pediatrneurol.2020.04.018","mag":"3023933160","pmid":"https://pubmed.ncbi.nlm.nih.gov/32684372"},"language":"en","primary_location":{"id":"doi:10.1016/j.pediatrneurol.2020.04.018","doi":"https://doi.org/10.1016/j.pediatrneurol.2020.04.018","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.pediatrneurol.2020.04.018","pdf_url":null,"source":{"id":"https://openalex.org/S165686931","display_name":"Pediatric Neurology","issn_l":"0887-8994","issn":["0887-8994","1873-5150"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-70","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Pediatric Neurology","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"hybrid","oa_url":"https://doi.org/10.1016/j.pediatrneurol.2020.04.018","any_repository_has_fulltext":false},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5065840657","display_name":"Emily de los Reyes","orcid":"https://orcid.org/0000-0002-1123-6903","observed_orcids":["https://orcid.org/0000-0002-1123-6903"]},"institutions":[{"id":"https://openalex.org/I1314135232","display_name":"Nationwide Children's Hospital","ror":"https://ror.org/003rfsp33","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1314135232"]},{"id":"https://openalex.org/I52357470","display_name":"The Ohio State University","ror":"https://ror.org/00rs6vg23","country_code":"US","type":"education","lineage":["https://openalex.org/I52357470"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Emily de los Reyes","raw_affiliation_strings":["Department of Pediatric Neurology, Nationwide Children's Hospital, The Ohio State University, Columbus, Ohio. Electronic address: Emily.delosReyes@nationwidechildrens.org","Department of Pediatric Neurology, Nationwide Children's Hospital, The Ohio State University, Columbus, Ohio"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pediatric Neurology, Nationwide Children's Hospital, The Ohio State University, Columbus, Ohio. Electronic address: Emily.delosReyes@nationwidechildrens.org","institution_ids":["https://openalex.org/I1314135232","https://openalex.org/I52357470"]},{"raw_affiliation_string":"Department of Pediatric Neurology, Nationwide Children's Hospital, The Ohio State University, Columbus, Ohio","institution_ids":["https://openalex.org/I1314135232","https://openalex.org/I52357470"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5020517973","display_name":"Lenora M. Lehwald","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1314135232","display_name":"Nationwide Children's Hospital","ror":"https://ror.org/003rfsp33","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1314135232"]},{"id":"https://openalex.org/I52357470","display_name":"The Ohio State University","ror":"https://ror.org/00rs6vg23","country_code":"US","type":"education","lineage":["https://openalex.org/I52357470"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Lenora Lehwald","raw_affiliation_strings":["Department of Pediatric Neurology, Nationwide Children's Hospital, The Ohio State University, Columbus, Ohio"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pediatric Neurology, Nationwide Children's Hospital, The Ohio State University, Columbus, Ohio","institution_ids":["https://openalex.org/I1314135232","https://openalex.org/I52357470"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5069764549","display_name":"Erika Fullwood Augustine","orcid":"https://orcid.org/0000-0001-7257-8985","observed_orcids":["https://orcid.org/0000-0001-7257-8985"]},"institutions":[{"id":"https://openalex.org/I28792581","display_name":"University of Rochester Medicine","ror":"https://ror.org/00trqv719","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I28792581","https://openalex.org/I5388228"]},{"id":"https://openalex.org/I5388228","display_name":"University of Rochester","ror":"https://ror.org/022kthw22","country_code":"US","type":"education","lineage":["https://openalex.org/I5388228"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Erika F. Augustine","raw_affiliation_strings":["Department of Neurology, University of Rochester Medical Center, Rochester, New York; Department of Pediatrics, University of Rochester Medical Center, Rochester, New York","Department of Neurology, University of Rochester Medical Center, Rochester, New York","Department of Pediatrics, University of Rochester Medical Center, Rochester, New York"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology, University of Rochester Medical Center, Rochester, New York; Department of Pediatrics, University of Rochester Medical Center, Rochester, New York","institution_ids":["https://openalex.org/I28792581","https://openalex.org/I5388228"]},{"raw_affiliation_string":"Department of Neurology, University of Rochester Medical Center, Rochester, New York","institution_ids":["https://openalex.org/I28792581","https://openalex.org/I5388228"]},{"raw_affiliation_string":"Department of Pediatrics, University of Rochester Medical Center, Rochester, New York","institution_ids":["https://openalex.org/I28792581","https://openalex.org/I5388228"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5016953572","display_name":"Elizabeth Berry\u2010Kravis","orcid":"https://orcid.org/0000-0001-7099-1522","observed_orcids":["https://orcid.org/0000-0001-7099-1522"]},"institutions":[{"id":"https://openalex.org/I1285301757","display_name":"Rush University Medical Center","ror":"https://ror.org/01j7c0b24","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1285301757"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Elizabeth Berry-Kravis","raw_affiliation_strings":["Department of Pediatrics, Rush University Medical Center, Chicago, Illinois; Department of Neurological Sciences, Rush University Medical Center, Chicago, Illinois","Department of Neurological Sciences, Rush University Medical Center, Chicago, Illinois","Department of Pediatrics, Rush University Medical Center, Chicago, Illinois"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pediatrics, Rush University Medical Center, Chicago, Illinois; Department of Neurological Sciences, Rush University Medical Center, Chicago, Illinois","institution_ids":["https://openalex.org/I1285301757"]},{"raw_affiliation_string":"Department of Neurological Sciences, Rush University Medical Center, Chicago, Illinois","institution_ids":["https://openalex.org/I1285301757"]},{"raw_affiliation_string":"Department of Pediatrics, Rush University Medical Center, Chicago, Illinois","institution_ids":["https://openalex.org/I1285301757"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5088928139","display_name":"Karen Butler","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I160606119","display_name":"University of Tennessee Health Science Center","ror":"https://ror.org/0011qv509","country_code":"US","type":"education","lineage":["https://openalex.org/I160606119"]},{"id":"https://openalex.org/I4210156806","display_name":"Le Bonheur Children's Hospital","ror":"https://ror.org/056wg8a82","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210156806"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Karen Butler","raw_affiliation_strings":["Neuroscience Institute, Le Bonheur Children's Hospital, Memphis, Tennessee; Division of Pediatric Neurology, University of Tennessee Health Science Center, Memphis, Tennessee","Division of Pediatric Neurology, University of Tennessee Health Science Center, Memphis, Tennessee","Neuroscience Institute, Le Bonheur Children's Hospital, Memphis, Tennessee"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Neuroscience Institute, Le Bonheur Children's Hospital, Memphis, Tennessee; Division of Pediatric Neurology, University of Tennessee Health Science Center, Memphis, Tennessee","institution_ids":["https://openalex.org/I160606119","https://openalex.org/I4210156806"]},{"raw_affiliation_string":"Division of Pediatric Neurology, University of Tennessee Health Science Center, Memphis, Tennessee","institution_ids":["https://openalex.org/I160606119"]},{"raw_affiliation_string":"Neuroscience Institute, Le Bonheur Children's Hospital, Memphis, Tennessee","institution_ids":["https://openalex.org/I4210156806"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5070027739","display_name":"Natalie Cormier","orcid":"https://orcid.org/0000-0002-3301-8771","observed_orcids":["https://orcid.org/0000-0002-3301-8771"]},"institutions":[{"id":"https://openalex.org/I4210147586","display_name":"Texas Children's Hospital","ror":"https://ror.org/05cz92x43","country_code":"US","type":"funder","lineage":["https://openalex.org/I4210147586"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Natalie Cormier","raw_affiliation_strings":["Department of Pediatric Neurosurgery, Texas Children's Hospital, Houston, Texas"],"raw_orcid":"https://orcid.org/0000-0002-3301-8771","affiliations":[{"raw_affiliation_string":"Department of Pediatric Neurosurgery, Texas Children's Hospital, Houston, Texas","institution_ids":["https://openalex.org/I4210147586"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5067016209","display_name":"Scott Demarest","orcid":"https://orcid.org/0000-0002-4544-3724","observed_orcids":["https://orcid.org/0000-0002-4544-3724"]},"institutions":[{"id":"https://openalex.org/I4210096275","display_name":"Children's Hospital Colorado","ror":"https://ror.org/00mj9k629","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210096275"]},{"id":"https://openalex.org/I51713134","display_name":"University of Colorado Anschutz","ror":"https://ror.org/03wmf1y16","country_code":"US","type":"education","lineage":["https://openalex.org/I51713134"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Scott Demarest","raw_affiliation_strings":["Departments of Pediatrics and Neurology, Children's Hospital Colorado, University of Colorado School of Medicine, Denver, Colorado"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Departments of Pediatrics and Neurology, Children's Hospital Colorado, University of Colorado School of Medicine, Denver, Colorado","institution_ids":["https://openalex.org/I4210096275","https://openalex.org/I51713134"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5040034837","display_name":"Sam Lu","orcid":"https://orcid.org/0000-0002-4754-7914","observed_orcids":["https://orcid.org/0000-0002-4754-7914"]},"institutions":[{"id":"https://openalex.org/I180670191","display_name":"University of California, San Francisco","ror":"https://ror.org/043mz5j54","country_code":"US","type":"education","lineage":["https://openalex.org/I180670191"]},{"id":"https://openalex.org/I4210135903","display_name":"UCSF Benioff Children's Hospital","ror":"https://ror.org/03hwe2705","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210135903"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Sam Lu","raw_affiliation_strings":["Department of Gastroenterology, UCSF Benioff Children's Hospital Oakland, Oakland, California"],"raw_orcid":"https://orcid.org/0000-0002-4754-7914","affiliations":[{"raw_affiliation_string":"Department of Gastroenterology, UCSF Benioff Children's Hospital Oakland, Oakland, California","institution_ids":["https://openalex.org/I180670191","https://openalex.org/I4210135903"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5112484129","display_name":"Jacqueline Madden","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I180670191","display_name":"University of California, San Francisco","ror":"https://ror.org/043mz5j54","country_code":"US","type":"education","lineage":["https://openalex.org/I180670191"]},{"id":"https://openalex.org/I4210135903","display_name":"UCSF Benioff Children's Hospital","ror":"https://ror.org/03hwe2705","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210135903"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Jacqueline Madden","raw_affiliation_strings":["Department of Gastroenterology, UCSF Benioff Children's Hospital Oakland, Oakland, California"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Gastroenterology, UCSF Benioff Children's Hospital Oakland, Oakland, California","institution_ids":["https://openalex.org/I180670191","https://openalex.org/I4210135903"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5032655470","display_name":"Joffre E. Olaya","orcid":"https://orcid.org/0000-0002-2039-6490","observed_orcids":["https://orcid.org/0000-0002-2039-6490"]},"institutions":[{"id":"https://openalex.org/I1304278710","display_name":"Children's Hospital of Orange County","ror":"https://ror.org/0282qcz50","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1304278710"]},{"id":"https://openalex.org/I204250578","display_name":"University of California, Irvine","ror":"https://ror.org/04gyf1771","country_code":"US","type":"education","lineage":["https://openalex.org/I204250578"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Joffre Olaya","raw_affiliation_strings":["Neuroscience Unit, Children's Hospital of Orange County, Orange, California; Department of Neurosurgery, University of California, Irvine School of Medicine, Orange, California","Department of Neurosurgery, University of California, Irvine School of Medicine, Orange, California","Neuroscience Unit, Children's Hospital of Orange County, Orange, California"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Neuroscience Unit, Children's Hospital of Orange County, Orange, California; Department of Neurosurgery, University of California, Irvine School of Medicine, Orange, California","institution_ids":["https://openalex.org/I1304278710","https://openalex.org/I204250578"]},{"raw_affiliation_string":"Department of Neurosurgery, University of California, Irvine School of Medicine, Orange, California","institution_ids":["https://openalex.org/I204250578"]},{"raw_affiliation_string":"Neuroscience Unit, Children's Hospital of Orange County, Orange, California","institution_ids":["https://openalex.org/I1304278710"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5052727392","display_name":"Susan See","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1304278710","display_name":"Children's Hospital of Orange County","ror":"https://ror.org/0282qcz50","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1304278710"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Susan See","raw_affiliation_strings":["Children's Hospital of Orange County, Orange, California"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Children's Hospital of Orange County, Orange, California","institution_ids":["https://openalex.org/I1304278710"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5059248455","display_name":"Amy Vierhile","orcid":"https://orcid.org/0000-0003-1821-2696","observed_orcids":["https://orcid.org/0000-0003-1821-2696"]},"institutions":[{"id":"https://openalex.org/I28792581","display_name":"University of Rochester Medicine","ror":"https://ror.org/00trqv719","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I28792581","https://openalex.org/I5388228"]},{"id":"https://openalex.org/I5388228","display_name":"University of Rochester","ror":"https://ror.org/022kthw22","country_code":"US","type":"education","lineage":["https://openalex.org/I5388228"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Amy Vierhile","raw_affiliation_strings":["Department of Neurology, University of Rochester Medical Center, Rochester, New York; Department of Pediatrics, University of Rochester Medical Center, Rochester, New York","Department of Neurology, University of Rochester Medical Center, Rochester, New York","Department of Pediatrics, University of Rochester Medical Center, Rochester, New York"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology, University of Rochester Medical Center, Rochester, New York; Department of Pediatrics, University of Rochester Medical Center, Rochester, New York","institution_ids":["https://openalex.org/I28792581","https://openalex.org/I5388228"]},{"raw_affiliation_string":"Department of Neurology, University of Rochester Medical Center, Rochester, New York","institution_ids":["https://openalex.org/I28792581","https://openalex.org/I5388228"]},{"raw_affiliation_string":"Department of Pediatrics, University of Rochester Medical Center, Rochester, New York","institution_ids":["https://openalex.org/I28792581","https://openalex.org/I5388228"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5032129336","display_name":"James W. Wheless","orcid":"https://orcid.org/0000-0002-4735-3431","observed_orcids":["https://orcid.org/0000-0002-4735-3431"]},"institutions":[{"id":"https://openalex.org/I160606119","display_name":"University of Tennessee Health Science Center","ror":"https://ror.org/0011qv509","country_code":"US","type":"education","lineage":["https://openalex.org/I160606119"]},{"id":"https://openalex.org/I4210156806","display_name":"Le Bonheur Children's Hospital","ror":"https://ror.org/056wg8a82","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210156806"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"James W. Wheless","raw_affiliation_strings":["Neuroscience Institute, Le Bonheur Children's Hospital, Memphis, Tennessee; Division of Pediatric Neurology, University of Tennessee Health Science Center, Memphis, Tennessee","Division of Pediatric Neurology, University of Tennessee Health Science Center, Memphis, Tennessee","Neuroscience Institute, Le Bonheur Children's Hospital, Memphis, Tennessee"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Neuroscience Institute, Le Bonheur Children's Hospital, Memphis, Tennessee; Division of Pediatric Neurology, University of Tennessee Health Science Center, Memphis, Tennessee","institution_ids":["https://openalex.org/I160606119","https://openalex.org/I4210156806"]},{"raw_affiliation_string":"Division of Pediatric Neurology, University of Tennessee Health Science Center, Memphis, Tennessee","institution_ids":["https://openalex.org/I160606119"]},{"raw_affiliation_string":"Neuroscience Institute, Le Bonheur Children's Hospital, Memphis, Tennessee","institution_ids":["https://openalex.org/I4210156806"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5027654915","display_name":"Amy Yang","orcid":"https://orcid.org/0000-0002-0196-6136","observed_orcids":["https://orcid.org/0000-0002-0196-6136"]},"institutions":[{"id":"https://openalex.org/I165690674","display_name":"Oregon Health & Science University","ror":"https://ror.org/009avj582","country_code":"US","type":"education","lineage":["https://openalex.org/I165690674"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Amy Yang","raw_affiliation_strings":["Department of Molecular and Medical Genetics, Oregon Health and Science University, Portland, Oregon"],"raw_orcid":"https://orcid.org/0000-0002-0196-6136","affiliations":[{"raw_affiliation_string":"Department of Molecular and Medical Genetics, Oregon Health and Science University, Portland, Oregon","institution_ids":["https://openalex.org/I165690674"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5042361960","display_name":"Jessica L. Cohen-Pfeffer","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I91786662","display_name":"BioMarin (United States)","ror":"https://ror.org/03fmvqd28","country_code":"US","type":"company","lineage":["https://openalex.org/I91786662"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Jessica Cohen-Pfeffer","raw_affiliation_strings":["BioMarin Pharmaceutical Inc., Novato, California"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"BioMarin Pharmaceutical Inc., Novato, California","institution_ids":["https://openalex.org/I91786662"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5004690450","display_name":"Dorna Chu","orcid":"https://orcid.org/0000-0002-1831-3377","observed_orcids":["https://orcid.org/0000-0002-1831-3377"]},"institutions":[{"id":"https://openalex.org/I91786662","display_name":"BioMarin (United States)","ror":"https://ror.org/03fmvqd28","country_code":"US","type":"company","lineage":["https://openalex.org/I91786662"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Dorna Chu","raw_affiliation_strings":["BioMarin Pharmaceutical Inc., Novato, California"],"raw_orcid":"https://orcid.org/0000-0002-1831-3377","affiliations":[{"raw_affiliation_string":"BioMarin Pharmaceutical Inc., Novato, California","institution_ids":["https://openalex.org/I91786662"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5048729566","display_name":"Fernanda Leal\u2010Pardinas","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I91786662","display_name":"BioMarin (United States)","ror":"https://ror.org/03fmvqd28","country_code":"US","type":"company","lineage":["https://openalex.org/I91786662"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Fernanda Leal-Pardinas","raw_affiliation_strings":["BioMarin Pharmaceutical Inc., Novato, California"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"BioMarin Pharmaceutical Inc., Novato, California","institution_ids":["https://openalex.org/I91786662"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5028989065","display_name":"Raymond Wang","orcid":"https://orcid.org/0000-0001-6494-7613","observed_orcids":["https://orcid.org/0000-0001-6494-7613"]},"institutions":[{"id":"https://openalex.org/I1304278710","display_name":"Children's Hospital of Orange County","ror":"https://ror.org/0282qcz50","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1304278710"]},{"id":"https://openalex.org/I204250578","display_name":"University of California, Irvine","ror":"https://ror.org/04gyf1771","country_code":"US","type":"education","lineage":["https://openalex.org/I204250578"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Raymond Y. Wang","raw_affiliation_strings":["Department of Pediatrics, Irvine School of Medicine, University of California, Orange, California; Department of Metabolic Disorders, Children's Hospital of Orange County, CHOC Children's Specialists, Orange, California","Department of Metabolic Disorders, Children's Hospital of Orange County, CHOC Children's Specialists, Orange, California","Department of Pediatrics, Irvine School of Medicine, University of California, Orange, California"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pediatrics, Irvine School of Medicine, University of California, Orange, California; Department of Metabolic Disorders, Children's Hospital of Orange County, CHOC Children's Specialists, Orange, California","institution_ids":["https://openalex.org/I1304278710"]},{"raw_affiliation_string":"Department of Metabolic Disorders, Children's Hospital of Orange County, CHOC Children's Specialists, Orange, California","institution_ids":["https://openalex.org/I1304278710"]},{"raw_affiliation_string":"Department of Pediatrics, Irvine School of Medicine, University of California, Orange, California","institution_ids":["https://openalex.org/I204250578"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":16,"corresponding_author_ids":["https://openalex.org/A5065840657"],"corresponding_institution_ids":["https://openalex.org/I1314135232","https://openalex.org/I52357470"],"apc_list":{"value":2700,"currency":"USD","value_usd":2700},"apc_paid":{"value":2700,"currency":"USD","value_usd":2700},"fwci":1.748,"has_fulltext":false,"cited_by_count":28,"citation_normalized_percentile":{"value":0.83895551,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":93,"max":99},"biblio":{"volume":"110","issue":null,"first_page":"64","last_page":"70"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10945","display_name":"Lysosomal Storage Disorders Research","score":0.9994999766349792,"subfield":{"id":"https://openalex.org/subfields/2737","display_name":"Physiology (medical)"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},"topics":[{"id":"https://openalex.org/T10945","display_name":"Lysosomal Storage Disorders Research","score":0.9994999766349792,"subfield":{"id":"https://openalex.org/subfields/2737","display_name":"Physiology (medical)"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T11027","display_name":"Metabolism and Genetic Disorders","score":9.999999747378752e-05,"subfield":{"id":"https://openalex.org/subfields/1308","display_name":"Clinical Biochemistry"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T14415","display_name":"Biomedical Research and Pathophysiology","score":9.999999747378752e-05,"subfield":{"id":"https://openalex.org/subfields/2734","display_name":"Pathology and Forensic Medicine"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/cerliponase-alfa","display_name":"cerliponase alfa","score":0.8830000162124634},{"id":"https://openalex.org/keywords/neuronal-ceroid-lipofuscinosis-type-2","display_name":"neuronal ceroid lipofuscinosis type 2","score":0.9639999866485596},{"id":"https://openalex.org/keywords/cln2-disease","display_name":"CLN2 disease","score":0.9269999861717224},{"id":"https://openalex.org/keywords/intracerebroventricular-infusion","display_name":"intracerebroventricular infusion","score":0.8880000114440918},{"id":"https://openalex.org/keywords/enzyme-replacement-therapy","display_name":"enzyme replacement therapy","score":0.9620000123977661},{"id":"https://openalex.org/keywords/lysosomal-storage-disorders","display_name":"lysosomal storage disease","score":0.9100000262260437},{"id":"https://openalex.org/keywords/central-nervous-system-infection","display_name":"central nervous system infection","score":0.8399999737739563},{"id":"https://openalex.org/keywords/cerebrospinal-fluid-culture","display_name":"cerebrospinal fluid culture","score":0.9049999713897705},{"id":"https://openalex.org/keywords/infection-control","display_name":"infection control","score":0.4909999966621399},{"id":"https://openalex.org/keywords/aseptic-technique","display_name":"aseptic technique","score":0.7509999871253967},{"id":"https://openalex.org/keywords/multidisciplinary-care","display_name":"multidisciplinary care","score":0.8119999766349792},{"id":"https://openalex.org/keywords/pediatric-neurodegenerative-disease","display_name":"pediatric neurodegenerative disease","score":0.6729999780654907}],"concepts":[{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.749526858329773},{"id":"https://openalex.org/C2779651940","wikidata":"https://www.wikidata.org/wiki/Q54196","display_name":"Cerebrospinal fluid","level":2,"score":0.5974305868148804},{"id":"https://openalex.org/C2779969927","wikidata":"https://www.wikidata.org/wiki/Q916766","display_name":"Enzyme replacement therapy","level":3,"score":0.5541149377822876},{"id":"https://openalex.org/C177713679","wikidata":"https://www.wikidata.org/wiki/Q679690","display_name":"Intensive care medicine","level":1,"score":0.46957680583000183},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.4568406641483307},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.29650843143463135}],"mesh":[{"descriptor_ui":"D000091346","descriptor_name":"Tripeptidyl-Peptidase 1","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000626","descriptor_name":"Aminopeptidases","qualifier_ui":"Q000172","qualifier_name":"deficiency","is_major_topic":true},{"descriptor_ui":"D002648","descriptor_name":"Child","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004152","descriptor_name":"Dipeptidyl-Peptidases and Tripeptidyl-Peptidases","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":true},{"descriptor_ui":"D004152","descriptor_name":"Dipeptidyl-Peptidases and Tripeptidyl-Peptidases","qualifier_ui":"Q000172","qualifier_name":"deficiency","is_major_topic":true},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007260","descriptor_name":"Infusion Pumps","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D007260","descriptor_name":"Infusion Pumps","qualifier_ui":"Q000592","qualifier_name":"standards","is_major_topic":true},{"descriptor_ui":"D009472","descriptor_name":"Neuronal Ceroid-Lipofuscinoses","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true},{"descriptor_ui":"D010348","descriptor_name":"Patient Care Team","qualifier_ui":"Q000592","qualifier_name":"standards","is_major_topic":true},{"descriptor_ui":"D011994","descriptor_name":"Recombinant Proteins","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":true},{"descriptor_ui":"D014481","descriptor_name":"United States","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D017410","descriptor_name":"Practice Guidelines as Topic","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D019635","descriptor_name":"Neurosurgical Procedures","qualifier_ui":"Q000592","qualifier_name":"standards","is_major_topic":true},{"descriptor_ui":"D033183","descriptor_name":"Interdisciplinary Communication","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D057057","descriptor_name":"Serine Proteases","qualifier_ui":"Q000172","qualifier_name":"deficiency","is_major_topic":true},{"descriptor_ui":"D057967","descriptor_name":"Infusions, Intraventricular","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true}],"locations_count":2,"locations":[{"id":"doi:10.1016/j.pediatrneurol.2020.04.018","doi":"https://doi.org/10.1016/j.pediatrneurol.2020.04.018","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.pediatrneurol.2020.04.018","pdf_url":null,"source":{"id":"https://openalex.org/S165686931","display_name":"Pediatric Neurology","issn_l":"0887-8994","issn":["0887-8994","1873-5150"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-70","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Pediatric Neurology","raw_type":"journal-article"},{"id":"pmid:32684372","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/32684372","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Pediatric neurology","raw_type":"Journal Article"}],"best_oa_location":{"id":"doi:10.1016/j.pediatrneurol.2020.04.018","doi":"https://doi.org/10.1016/j.pediatrneurol.2020.04.018","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.pediatrneurol.2020.04.018","pdf_url":null,"source":{"id":"https://openalex.org/S165686931","display_name":"Pediatric Neurology","issn_l":"0887-8994","issn":["0887-8994","1873-5150"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-70","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Pediatric Neurology","raw_type":"journal-article"},"sustainable_development_goals":[{"score":1.0,"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being"}],"study_designs":[],"awards":[],"funders":[{"id":"https://openalex.org/F4320310557","display_name":"BioMarin Pharmaceutical","ror":"https://ror.org/03fmvqd28"}],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":29,"referenced_works":["https://openalex.org/W1602997642","https://openalex.org/W1972488241","https://openalex.org/W2010224249","https://openalex.org/W2019662203","https://openalex.org/W2027013896","https://openalex.org/W2082014903","https://openalex.org/W2112479854","https://openalex.org/W2113010344","https://openalex.org/W2148670386","https://openalex.org/W2314293318","https://openalex.org/W2339879146","https://openalex.org/W2411939482","https://openalex.org/W2497239520","https://openalex.org/W2506977751","https://openalex.org/W2508990490","https://openalex.org/W2550801800","https://openalex.org/W2583565371","https://openalex.org/W2588163058","https://openalex.org/W2622228475","https://openalex.org/W2799850385","https://openalex.org/W2803829393","https://openalex.org/W2811299944","https://openalex.org/W2884337412","https://openalex.org/W2977490654","https://openalex.org/W3021422704","https://openalex.org/W4321429426","https://openalex.org/W6655608559","https://openalex.org/W6753442750","https://openalex.org/W6758487896"],"related_works":["https://openalex.org/W7204989480","https://openalex.org/W7138950496","https://openalex.org/W4413471877","https://openalex.org/W4411177883","https://openalex.org/W4388373503","https://openalex.org/W2997482341","https://openalex.org/W3128665212","https://openalex.org/W2799850385","https://openalex.org/W3110614618","https://openalex.org/W3211280224"],"abstract_inverted_index":{"BACKGROUND:":[0],"Neuronal":[1],"ceroid":[2],"lipofuscinosis":[3],"type":[4],"2":[5],"or":[6],"CLN2":[7,41,73],"disease":[8,42,74],"is":[9,33,148,161,211],"a":[10,26,107,149,188,197],"rare,":[11],"autosomal":[12],"recessive,":[13],"neurodegenerative":[14],"lysosomal":[15],"storage":[16],"disorder":[17],"caused":[18],"by":[19,222],"tripeptidyl":[20,29],"peptidase":[21,30],"1":[22,31],"deficiency.":[23],"Cerliponase":[24],"alfa,":[25],"recombinant":[27],"human":[28],"enzyme,":[32],"the":[34,44,89,171,185,201],"first":[35,45],"and":[36,43,85,113,115,127,130,200,218,245],"only":[37],"approved":[38,46],"treatment":[39,69],"for":[40,156,242],"enzyme":[47],"replacement":[48],"therapy":[49],"administered":[50],"via":[51],"intracerebroventricular":[52,137,146,202],"infusion.":[53,133],"METHODS:":[54],"A":[55],"meeting":[56],"of":[57,70,91,99,110,136,173,237],"health":[58],"care":[59,247],"professionals":[60],"from":[61],"US":[62],"institutions":[63,177,223,231],"with":[64,72,168,224],"experience":[65,227],"in":[66,77,152,170,232],"cerliponase":[67,100,225],"alfa":[68,226],"children":[71],"was":[75],"held":[76],"November":[78],"2018.":[79],"Key":[80,103],"common":[81,216],"practices":[82,104,217,220],"were":[83],"identified,":[84],"later":[86],"refined":[87],"during":[88,132,144],"drafting":[90],"this":[92],"article,":[93],"that":[94],"facilitate":[95],"safe":[96],"chronic":[97],"administration":[98],"alfa.":[101],"RESULTS:":[102],"include":[105],"developing":[106,243],"multidisciplinary":[108],"team":[109],"clinicians,":[111],"pharmacists,":[112],"coordinators,":[114],"institution-specific":[116],"processes.":[117],"Infection":[118],"risk":[119],"may":[120,228],"be":[121,194,205,240],"reduced":[122],"through":[123],"strict":[124],"aseptic":[125],"techniques":[126],"minimizing":[128],"connections":[129],"disconnections":[131],"The":[134,214],"impact":[135],"device":[138,153,203],"design":[139],"on":[140,196],"port":[141],"needle":[142],"stability":[143],"extended":[145],"infusion":[147],"critical":[150],"consideration":[151],"selection.":[154],"Monitoring":[155],"central":[157],"nervous":[158],"system":[159],"infection":[160,210],"performed":[162],"at":[163],"each":[164],"patient":[165,246],"contact,":[166],"but":[167],"flexibility":[169],"degree":[172],"monitoring.":[174],"Although":[175],"few":[176],"had":[178],"experienced":[179],"positive":[180,189],"cerebrospinal":[181,190,208],"fluid":[182,191,209],"test":[183],"results,":[184],"response":[186],"to":[187],"culture":[192],"should":[193,204],"determined":[195],"case-by-case":[198],"basis,":[199],"removed":[206],"if":[207],"confirmed.":[212],"CONCLUSIONS:":[213],"key":[215],"flexible":[219],"used":[221],"assist":[229],"other":[230],"process":[233],"development.":[234],"Continued":[235],"sharing":[236],"experiences":[238],"will":[239],"essential":[241],"standards":[244],"guidelines.":[248]},"counts_by_year":[{"year":2026,"cited_by_count":7},{"year":2025,"cited_by_count":5},{"year":2024,"cited_by_count":4},{"year":2023,"cited_by_count":3},{"year":2022,"cited_by_count":4},{"year":2021,"cited_by_count":2},{"year":2020,"cited_by_count":3}],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2020-05-13T00:00:00"}
