المؤلفون: Christine Haller, Wenjie Song, Tricia Cimms, Chao‐Yin Chen, Chester B. Whitley, Raymond Y. Wang, Mislen Bauer, Paul Harmatz

المصدر: JIMD Reports, Vol 49, Iss 1, Pp 53-62 (2019)

مصطلحات موضوعية: enzyme replacement therapy, heat map, MPS VII, mucopolysaccharidosis, Sly syndrome, vestronidase alfa, Diseases of the endocrine glands. Clinical endocrinology, RC648-665, Genetics, QH426-470

وصف الملف: electronic resource

Relation: https://doaj.org/toc/2192-8312

URL الوصول: https://doaj.org/article/45171029c10d4a88ae6609f3c07fd284

المؤلفون: Paul Harmatz, Tricia Cimms, Wenjie Song, Raymond Y. Wang, Christine Haller, Mislen Bauer, Chester B. Whitley, Chao Yin Chen

المصدر: JIMD Reports, Vol 49, Iss 1, Pp 53-62 (2019)
JIMD reports, vol 49, iss 1
JIMD Reports

مصطلحات موضوعية: Research Report, medicine.medical_specialty, Randomization, Mucopolysaccharidoses (MPS), lcsh:QH426-470, Endocrinology, Diabetes and Metabolism, Mucopolysaccharidosis, Clinical Trials and Supportive Activities, vestronidase alfa, Mucopolysaccharidosis VII, Disease, MPS VII, heat map, Biochemistry, Genetics and Molecular Biology (miscellaneous), lcsh:Diseases of the endocrine glands. Clinical endocrinology, 03 medical and health sciences, 0302 clinical medicine, Rare Diseases, Quality of life, Clinical Research, Behavioral and Social Science, Internal Medicine, Clinical endpoint, Medicine, Pediatric, 0303 health sciences, lcsh:RC648-665, business.industry, 030305 genetics & heredity, Research Reports, mucopolysaccharidosis, Enzyme replacement therapy, medicine.disease, 3. Good health, lcsh:Genetics, Sly syndrome, Physical therapy, Population study, business, 030217 neurology & neurosurgery, enzyme replacement therapy

وصف الملف: application/pdf

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::57f686c939e50c42430cfe653bdda633
https://doaj.org/article/45171029c10d4a88ae6609f3c07fd284

المؤلفون: Jack Shi, Julie Taylor, Wenjie Song, Kathleen McKeever, Yulan Qi, Simon Jones, Christine Haller

المصدر: Clinical Pharmacokinetics

مصطلحات موضوعية: 0301 basic medicine, Adult, Male, Adolescent, Mucopolysaccharidosis, Population, Sly syndrome, Pharmacology, Models, Biological, 03 medical and health sciences, Young Adult, 0302 clinical medicine, Pharmacokinetics, Medicine, Humans, Pharmacology (medical), Computer Simulation, Enzyme Replacement Therapy, Original Research Article, Dosing, education, Child, Glucuronidase, Glycosaminoglycans, education.field_of_study, Cross-Over Studies, business.industry, Mucopolysaccharidosis VII, Infant, Correction, Enzyme replacement therapy, medicine.disease, Crossover study, 030104 developmental biology, Pharmacodynamics, Child, Preschool, Female, business, 030217 neurology & neurosurgery

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::96d6939aed0f1f22037fe10295afefb5
https://pubmed.ncbi.nlm.nih.gov/30515718

المؤلفون: Christine Haller

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::babeba28e436852087968406faeacc6b
https://doi.org/10.26226/morressier.5b50866b6a42700010585c09

المؤلفون: Mislen Bauer, Paul Harmatz, Raymond Y. Wang, Emil D. Kakkis, Chester B. Whitley, Wenjie Song, Christine Haller

المصدر: Molecular genetics and metabolism. 123(4)

مصطلحات موضوعية: 0301 basic medicine, Adult, Male, medicine.medical_specialty, Vital capacity, Visual acuity, Adolescent, Endocrinology, Diabetes and Metabolism, Sly syndrome, Mucopolysaccharidosis VII, Disease, Biochemistry, 03 medical and health sciences, Young Adult, Endocrinology, Quality of life, Internal medicine, Genetics, Clinical endpoint, medicine, Humans, Child, Molecular Biology, Glucuronidase, business.industry, Enzyme replacement therapy, medicine.disease, Prognosis, Recombinant Proteins, 030104 developmental biology, Female, medicine.symptom, business, Follow-Up Studies

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::35e3c2b33f3183fdcb7f7aebef745b40
https://pubmed.ncbi.nlm.nih.gov/29478819

المؤلفون: Heather Lau, Christine Haller, Gregory M. Pastores, Vassili Valayannopoulos, Eugen Mengel, Matthew Mealiffe, Ke Yang, Derralynn Hughes, Roberto Giugliani, Rossella Parini, Paul Harmatz, Christian J. Hendriksz, Julian Raiman, Moeenaldeen Al-Sayed, Nathalie Guffon

المساهمون: Amsterdam Gastroenterology Endocrinology Metabolism, Paediatric Metabolic Diseases

المصدر: Molecular genetics and metabolism, 114(2), 178-185. Academic Press Inc.

مصطلحات موضوعية: Adult, medicine.medical_specialty, Adolescent, Idursulfase, Mucopolysaccharidosis, Endocrinology, Diabetes and Metabolism, Placebo, Biochemistry, law.invention, Young Adult, chemistry.chemical_compound, Endocrinology, Double-Blind Method, Randomized controlled trial, Elosulfase alfa, law, Surveys and Questionnaires, Internal medicine, Activities of Daily Living, medicine, Genetics, Humans, Enzyme Replacement Therapy, Respiratory function, Young adult, Child, Molecular Biology, business.industry, Mucopolysaccharidosis IV, Maximal Voluntary Ventilation, Middle Aged, medicine.disease, Body Height, Chondroitinsulfatases, Respiratory Function Tests, Treatment Outcome, chemistry, Child, Preschool, Physical therapy, Population study, business, medicine.drug

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d5f05ff6fc2019d2f08a79baf127f5e8
https://doi.org/10.1016/j.ymgme.2014.08.012

المؤلفون: Rob Arbuckle, Tricia Cimms, Qais Abu Ali, Christine Haller, Andrea Schatz, Chloe Johnson, Betsy Malkus, Sarah Kilgariff

المصدر: Molecular Genetics and Metabolism. 123:S32-S33

مصطلحات موضوعية: 0301 basic medicine, business.industry, Endocrinology, Diabetes and Metabolism, medicine.disease, Biochemistry, 03 medical and health sciences, 030104 developmental biology, Endocrinology, Genetics, medicine, Galactosialidosis, business, Molecular Biology, Qualitative research, Clinical psychology

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::79717a8943852349d7c66a870e3a2c17
https://doi.org/10.1016/j.ymgme.2017.12.063

المؤلفون: V. Reid Sutton, Qais Abu Ali, Christine Haller, Amanda Chanda, José Francisco da Silva Franco, Esmeralda Martins, Jaime López-Valdez, Chester B. Whitley, Wenjie Song, Raymond Y. Wang, Paul Harmatz

المصدر: Molecular Genetics and Metabolism. 123:S144-S145

مصطلحات موضوعية: 0301 basic medicine, medicine.medical_specialty, business.industry, Endocrinology, Diabetes and Metabolism, Vestronidase alfa, Enzyme replacement therapy, Biochemistry, Gastroenterology, 03 medical and health sciences, 030104 developmental biology, Endocrinology, Internal medicine, Genetics, medicine, In patient, business, Molecular Biology

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::7598dd4f2218e33a174e00f6fe7bee4c
https://doi.org/10.1016/j.ymgme.2017.12.400

المؤلفون: Yulan Qi, Kathleen McKeever, Julie Taylor, Christine Haller, Wenjie Song, Simon A. Jones, Jack Shi

المصدر: Clinical Pharmacokinetics. 58:685-685

مصطلحات موضوعية: Pharmacology, Pharmacology (medical)

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::84f33e2a292b9450cea6d611e75f61c4
https://doi.org/10.1007/s40262-018-0726-6

المؤلفون: Qais Abu Ali, Christine Haller, Sean Daugherty, Mislen Bauer, Chao Yin Chen, Wenjie Song, Raymond Y. Wang, Paul Harmatz, Tricia Cimms, Chester B. Whitley

المصدر: Molecular Genetics and Metabolism. 123:S15

مصطلحات موضوعية: Endocrinology, business.industry, Endocrinology, Diabetes and Metabolism, Immunology, Genetics, Population study, Medicine, Enzyme replacement therapy, business, Molecular Biology, Biochemistry, Genotype phenotype

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::0e44cee63d5efac77a08c84e6baf8370
https://doi.org/10.1016/j.ymgme.2017.12.011