Browse Items (4 total)

Tags: 1-Deoxynojirimycin/analogs & derivatives/therapeutic use, 2006, Backlog, Bone Marrow Transplantation, Brady RO, Enzyme Inhibitors/therapeutic use, Fabry Disease/therapy, Gangliosidosis, Gaucher Disease/therapy, Gene Therapy, GM1/therapy, Humans, Imino Sugars/therapeutic use, Journal Article, Longitudinal Studies, Piperidines/therapeutic use, Rejuvenation Research

Tags: 0 (Enzyme Inhibitors), 0 (Lamin Type A), 0 (Piperidines), 0 (prelamin A), 0 (Pyridines), 2018, Adolescent, Adult, Brazier J, Campbell SE, Cause Of Death, Child, Cohort Studies, D'Agostino Sr RB, EC 2.7.4.- (farnesyl phosphate kinase), EC 2.7.4.- (Phosphotransferases (Phosphate Group Acceptor)), Enzyme Inhibitors/therapeutic use, Female, Gordon LB, Humans, IOW153004F (lonafarnib), JAMA., July 2018 List, Kaplan-Meier Estimate, Kieran MW, Kleinman ME, Lamin Type A/biosynthesis/metabolism, Male, Massaro J, Phosphotransferases (Phosphate Group Acceptor)/antagonists & inhibitors, Piperidines/therapeutic use, Post-Translational, Progeria/drug therapy/genetics/mortality, Protein Processing, Pyridines/therapeutic use, Shappell H, Young Adult

Tags: 2007, Adolescent, Backlog, Child, Diagnosis, Diagnostic Techniques, Differential, Digestive System, Enzyme Inhibitors/therapeutic use, Gastroesophageal Reflux/complications/diagnosis/physiopathology/therapy, Histamine H2 Antagonists/therapeutic use, Humans, Infant, Journal Article, Medical History Taking/methods, Michail S, Nervous System Diseases/complications, Nutritional Failure, Pediatrics In Review / American Academy Of Pediatrics, Pediatrics/methods, Preschool, Prognosis, Proton Pumps/antagonists & inhibitors, Respiratory Tract Diseases/etiology/prevention & control, Vomiting/diagnosis/etiology

Tags: 1-Deoxynojirimycin/analogs & derivatives/therapeutic use, 2007, Abel L, Adolescent, Adult, Age Factors, Analysis of Variance, Backlog, Child, Cohort Studies, Deglutition/drug effects, Double-Blind Method, Drug Administration Schedule, Enzyme Inhibitors/therapeutic use, Female, Humans, Journal Article, Lancet Neurology, Male, Niemann-Pick Disease, Patterson MC, Prady H, Preschool, Q3 Scoping Review Results, Retrospective Studies, Saccades/drug effects, Severity Of Illness Index, Type C/drug therapy/physiopathology, Vecchio D, Wraith JE