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1دورية أكاديمية
المؤلفون: Li, Keying, Kim, Jeongtae, Jeong, Yujin, Lee, Moo-SeungAff2, Aff3, Koo, ChiwanAff1, IDs13206024001551_cor5
المصدر: BioChip Journal. 18(3):373-381
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2دورية أكاديمية
المؤلفون: Wanner, Christoph, Kimonis, Virginia, Politei, Juan, Warnock, David G, Üçeyler, Nurcan, Frey, Aline, Cornelisse, Peter, Hughes, Derralyn
مصطلحات موضوعية: Biomedical and Clinical Sciences, Clinical Sciences, Digestive Diseases, Pain Research, Neurodegenerative, Peripheral Neuropathy, Patient Safety, Clinical Research, Clinical Trials and Supportive Activities, Chronic Pain, Neurosciences, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, AE, adverse event, BPI-SF, Brief Pain Inventory-Short Form, BPI-SF3, Brief Pain Inventory-Short Form item 3, BSS, Bristol stool scale, CD, cognitive debriefing, CE, concept elicitation, CESD-R-20, Center for Epidemiologic Studies Depression Scale Revised, CKD-EPI, Chronic Kidney Disease Epidemiology Collaboration, CTCAE, Common Terminology Criteria for Adverse Events, ECG, electrocardiography, EOS, end of study, EOT, end-of-treatment, ERT, enzyme replacement therapy, FABPRO-GI, FABry Disease Patient-Reported Outcome-GastroIntestinal, FD, Fabry disease, FGID, functional gastrointestinal disorders, Fabry disease, GCS, glucosylceramide synthase, GI, gastrointestinal, GSRS, Gastrointestinal Symptom Rating Scale, Gb3, globotriaosylceramide, HbA1c, hemoglobin A1c, IBS, irritable bowel syndrome, IRB, independent review board, LVEF, left ventricular ejection fraction, LVMI, left ventricular mass index, Lucerastat, MODIFY, NPSI, neuropathic pain symptom inventory, NRS-11, 11-point numerical rating scale, NYHA, New York Heart Association, NeP, neuropathic pain, OLE, open-label extension, PGIC-DS, Patient Global Impression of Change in Disease Severity, PGIC-PS, Patient Global Impression of Change in neuropathic Pain Severity, PGIS-D, Patient Global Impression of Severity of Disease, PGIS-P, Patient Global Impression of Severity of neuropathic pain, PK, pharmacokinetics, PRO, patient-reported outcome, SD, standard deviation, SF-36v2, 36-Item Short Form Health Survey Version 2, SRT, substrate reduction therapy, Substrate reduction therapy, UCI, University of California, Irvine, UT, usability testing, b.i.d., twice daily, eGFR, estimated glomerular filtration rate, α-GAL A, lysosomal enzyme α-galactosidase, Biochemistry and Cell Biology, Genetics, Clinical sciences
وصف الملف: application/pdf
URL الوصول: https://escholarship.org/uc/item/5nx9f0kw
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3دورية أكاديمية
المؤلفون: Deng, Mulan, Zhou, Hongyu, He, Shaomei, Qiu, Haoheng, Wang, Yanping, Zhao, April Yuanyi, Mu, YunpingAff1, IDs13023023028940_cor7, Li, FanghongAff1, IDs13023023028940_cor8, Zhao, Allan ZijianAff1, IDs13023023028940_cor9
المصدر: Orphanet Journal of Rare Diseases. 18(1)
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4دورية أكاديمية
المؤلفون: Mulan Deng, Hongyu Zhou, Shaomei He, Haoheng Qiu, Yanping Wang, April Yuanyi Zhao, Yunping Mu, Fanghong Li, Allan Zijian Zhao
المصدر: Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-13 (2023)
مصطلحات موضوعية: Fabry disease (FD), Adeno-associated viral 2/8 (AAV2/8), α-galactosidase A (α-Gal A), Globotriaosylceramide (Gb3), Globotriaosylsphingosine (Lyso-Gb3), Lysosomal storage diseases (LSDs), Medicine
وصف الملف: electronic resource
Relation: https://doaj.org/toc/1750-1172
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5دورية أكاديمية
المؤلفون: Aranzazu Villasante, Josep Corominas, Clara Alcon, Andrea Garcia-Lizarribar, Jaume Mora, Monica Lopez-Fanarraga, Josep Samitier
المصدر: Cancers, Vol 16, Iss 5, p 1060 (2024)
مصطلحات موضوعية: GB3, neuroblastoma, alternative vasculature, tumor-derived endothelial cells, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282
وصف الملف: electronic resource
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6دورية أكاديمية
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7دورية أكاديمية
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8دورية أكاديمية
المؤلفون: Meléndez, Ana ValeriaAff1, Aff2, Aff3, Aff4, Velasco Cárdenas, Rubí M.-H.Aff1, Aff2, Aff3, Lagies, Simon, Strietz, Juliane, Siukstaite, LinaAff1, Aff2, Aff3, Thomas, Oliver S.Aff1, Aff2, Aff3, Aff4, Tomisch, JanaAff1, Aff2, Aff3, Weber, WilfriedAff1, Aff2, Aff3, Aff4, Kammerer, BerndAff2, Aff5, Aff7, Römer, WinfriedAff1, Aff2, Aff3, Aff4, Aff8, IDs00018022045247_cor10, Minguet, SusanaAff1, Aff2, Aff3, Aff4, Aff8, Aff9, IDs00018022045247_cor11
المصدر: Cellular and Molecular Life Sciences. 79(10)
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9دورية أكاديمية
المؤلفون: Beatriz San Millán-Tejado, Carmen Navarro, Julián Fernández-Martín, Alberto Rivera, Irene Viéitez, Susana Teijeira, Saida Ortolano
المصدر: Journal of Clinical Medicine, Vol 12, Iss 17, p 5689 (2023)
مصطلحات موضوعية: Fabry disease, Gb3, lysosome, biopsy, electron microscopy, Medicine
وصف الملف: electronic resource
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10دورية أكاديمية
المؤلفون: Marina Beraza-Millor, Julen Rodríguez-Castejón, Jonatan Miranda, Ana del Pozo-Rodríguez, Alicia Rodríguez-Gascón, María Ángeles Solinís
المصدر: Pharmaceutics, Vol 15, Iss 7, p 1936 (2023)
مصطلحات موضوعية: substrate reduction therapy, solid lipid nanoparticles, siRNA, Fabry disease, Gb3 synthase, gold nanoparticles, Pharmacy and materia medica, RS1-441
وصف الملف: electronic resource