نتائج البحث - "Cerliponase alfa"

المساهمون: Pediatrics, Orthopedics and Sports Medicine, Child and Adolescent Psychiatry / Psychology, Clinical Genetics, Radiology & Nuclear Medicine, Neurosurgery

المصدر: Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases, 16(1):221. BioMed Central Ltd.
Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-9 (2021)

مصطلحات موضوعية: 0301 basic medicine, Pediatrics, medicine.medical_specialty, Lysosomal storage disorder, Tripeptidyl peptidase, Presymptomatic, Cerliponase alfa, Disease, CLN2 disease, Bayley Scales of Infant Development, Cohort Studies, 03 medical and health sciences, 0302 clinical medicine, Neuronal Ceroid-Lipofuscinoses, Medicine, Humans, Pharmacology (medical), Toddler, Late-infantile neuronal ceroid lipofuscinosis, Dipeptidyl-Peptidases and Tripeptidyl-Peptidases, Genetics (clinical), Tripeptidyl-Peptidase 1, business.industry, Research, Infant, General Medicine, Enzyme replacement therapy, Recombinant Proteins, Neuronal Ceroid Lipofuscinosis Type 2, 030104 developmental biology, Child, Preschool, Intracerebroventricular, Evoked Potentials, Visual, Cerebellar atrophy, business, 030217 neurology & neurosurgery, Cohort study

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::826aa62f4a612dc16473ce736a138c8bTest
https://pubmed.ncbi.nlm.nih.gov/33990214Test

6

Current and Emerging Treatment Strategies for Neuronal Ceroid Lipofuscinoses

المؤلفون: Angela Schulz, Stephan Storch, Alfried Kohlschütter, Udo Bartsch

المصدر: CNS Drugs

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ee0101a5e15f7747e9cef88f5a0a98a5Test
https://doi.org/10.1007/s40263-019-00620-8Test

7

Immunogenicity to cerliponase alfa intracerebroventricular enzyme replacement therapy for CLN2 disease: Results from a Phase 1/2 study

المؤلفون: Becky Schweighardt, Temitayo Ajayi, Charles A. O'Neill, Joshua Henshaw, Greg de Hart, Heather Cahan, Anu Cherukuri, Andrea Van Tuyl, Dave Jacoby, Laurie Bray, Peter Slasor

المصدر: Clinical Immunology. 197:68-76

مصطلحات موضوعية: Male, 0301 basic medicine, medicine.medical_specialty, Adolescent, Immunology, Cerliponase alfa, Immunoglobulin E, Gastroenterology, Antibodies, Drug Hypersensitivity, 03 medical and health sciences, 0302 clinical medicine, Cerebrospinal fluid, Neuronal Ceroid-Lipofuscinoses, Internal medicine, medicine, Humans, Immunology and Allergy, Enzyme Replacement Therapy, Child, Dipeptidyl-Peptidases and Tripeptidyl-Peptidases, Adverse effect, Tripeptidyl-Peptidase 1, biology, business.industry, Immunogenicity, Enzyme replacement therapy, medicine.disease, Antibodies, Neutralizing, Recombinant Proteins, Infusions, Intraventricular, 030104 developmental biology, Child, Preschool, Disease Progression, biology.protein, Female, Antibody, business, 030217 neurology & neurosurgery, Anaphylaxis

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c9190f89b20d2a23077cb439a3658026Test
https://doi.org/10.1016/j.clim.2018.09.003Test

8

Disease characteristics and progression in patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease: an observational cohort study

المؤلفون: Temitayo Ajayi, Benjamin Van de Graaf, Ronald G. Crystal, Kaleb Yohay, Odelya E. Pagovich, Dolan Sondhi, Matthew Downs, Alessandro Simonati, David Jacoby, Peter Slasor, Susanne Lezius, Dirk Kilian, Angela Schulz, Miriam Nickel, Barry E. Kosofsky, Alfried Kohlschütter

المصدر: The Lancet Child & Adolescent Health. 2:582-590

مصطلحات موضوعية: Male, 0301 basic medicine, Pediatrics, medicine.medical_specialty, Adolescent, assessment, Cerliponase alfa, Disease, patients, Cohort Studies, 03 medical and health sciences, Epilepsy, 0302 clinical medicine, Neuronal Ceroid-Lipofuscinoses, Developmental and Educational Psychology, medicine, Humans, Dementia, Longitudinal Studies, Child, Psychomotor learning, disease, therapy, Tripeptidyl-Peptidase 1, business.industry, Late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2), progression, Infant, medicine.disease, Natural history, 030104 developmental biology, Child, Preschool, Pediatrics, Perinatology and Child Health, Cohort, Disease Progression, Female, business, 030217 neurology & neurosurgery, Cohort study

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::866bc0beced8cf0629c622f81a9fdbb8Test
https://doi.org/10.1016/s2352-4642Test(18)30179-2

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Cerliponase alfa changes the natural history of children with neuronal ceroid lipofuscinosis type 2: The first French cohort

المؤلفون: Celia Hoebeke, Didier Scavarda, Isabelle Desguerre, Stéphane Auvin, Aline Cano, Samia Pichard, Brigitte Chabrol, Bastien Estublier

المساهمون: Institut de Neurosciences des Systèmes (INS), Aix Marseille Université (AMU)-Institut National de la Santé et de la Recherche Médicale (INSERM)

المصدر: European Journal of Paediatric Neurology
European Journal of Paediatric Neurology, Elsevier, 2021, 30, pp.17-21. ⟨10.1016/j.ejpn.2020.12.002⟩

مصطلحات موضوعية: Male, Pediatrics, medicine.medical_specialty, Cerliponase alfa, Gene mutation, Time-to-Treatment, Cohort Studies, 03 medical and health sciences, 0302 clinical medicine, Neuronal Ceroid-Lipofuscinoses, 030225 pediatrics, medicine, Humans, Enzyme Replacement Therapy, Stage (cooking), Child, Dipeptidyl-Peptidases and Tripeptidyl-Peptidases, ComputingMilieux_MISCELLANEOUS, Retrospective Studies, Tripeptidyl-Peptidase 1, business.industry, [SCCO.NEUR]Cognitive science/Neuroscience, General Medicine, Enzyme replacement therapy, Tripeptidyl peptidase I, Recombinant Proteins, Natural history, Neuronal Ceroid Lipofuscinosis Type 2, Child, Preschool, Pediatrics, Perinatology and Child Health, Cohort, Disease Progression, Female, Neurology (clinical), France, business, 030217 neurology & neurosurgery

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::727f4e4c97bb167672eec8b5a0f0a2fdTest
https://pubmed.ncbi.nlm.nih.gov/33348105Test

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Study of Intraventricular Cerliponase Alfa for CLN2 Disease

المؤلفون: Angela, Schulz, Temitayo, Ajayi, Nicola, Specchio, Emily, de Los Reyes, Paul, Gissen, Douglas, Ballon, Jonathan P, Dyke, Heather, Cahan, Peter, Slasor, David, Jacoby, Alfried, Kohlschütter, Barbara, Csányi

المصدر: New England Journal of Medicine. 378:1898-1907

مصطلحات موضوعية: Male, 0301 basic medicine, medicine.medical_specialty, Adolescent, Progressive dementia, Kaplan-Meier Estimate, Disease, Cerliponase alfa, Language Development, Gastroenterology, Tripeptidyl peptidase, 03 medical and health sciences, 0302 clinical medicine, Neuronal Ceroid-Lipofuscinoses, Internal medicine, medicine, Humans, Dementia, Enzyme Replacement Therapy, Child, Dipeptidyl-Peptidases and Tripeptidyl-Peptidases, Tripeptidyl-Peptidase 1, business.industry, Historically Controlled Study, General Medicine, Enzyme replacement therapy, medicine.disease, Recombinant Proteins, Neuronal Ceroid Lipofuscinosis Type 2, Clinical trial, Infusions, Intraventricular, 030104 developmental biology, Motor Skills, Child, Preschool, Disease Progression, Female, business, 030217 neurology & neurosurgery

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d43c9da56520f991daaca69dc0323363Test
https://doi.org/10.1056/nejmoa1712649Test

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