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Journal of Pediatrics, The, ISSN 0022-3476, 2010, Volume 156, Issue 4, pp. 531 - 531
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 12/2011, Volume 365, Issue 25, pp. 2357 - 2365
Journal Article
Journal of Pediatrics, ISSN 0022-3476, 04/2010, Volume 156, Issue 4, p. 531
Journal Article
American Journal of Medical Genetics Part C: Seminars in Medical Genetics, ISSN 1552-4868, 03/2014, Volume 166, Issue 1, pp. 45 - 55
Journal Article
Expert Opinion on Biological Therapy, ISSN 1471-2598, 01/2016, Volume 16, Issue 1, pp. 79 - 92
Introduction: Hemophilia B is a sex linked, monogenic, coagulation disorder caused by a deficiency in functional factor IX protein. Gene therapy for this... 
gene delivery | factor IX | hemophilia | FIX | AAV | gene therapy | adeno-associated virus | coagulopathy | serotype | Hemophilia B - therapy | Dependovirus - genetics | Animals | Humans | Half-Life | Quality of Life | Factor IX - genetics | Genetic Vectors - therapeutic use | Hemophilia B - genetics | Transgenes | Genetic Vectors - genetics | Genetic Therapy - methods
Journal Article
Cochrane Database of Systematic Reviews, ISSN 1469-493X, 12/2016, Volume 2016, Issue 12, p. CD010822
Background Haemophilia is a genetic disorder characterized by spontaneous or provoked, often uncontrolled, bleeding into joints, muscles and other soft... 
Genetic Therapy | HUMAN-FACTOR-IX | Humans | FC FUSION PROTEIN | TRANSDUCTION | PROPHYLAXIS | MEDICINE, GENERAL & INTERNAL | DISEASE | LIVER | Hemophilia A [therapy] | VIRUS VECTOR INTEGRATION | MOLECULAR-CLONING | Hemophilia B [therapy] | COAGULATION-FACTOR VIII | LONG | Hemophilia B - therapy | Hemophilia A - therapy
Journal Article
Journal of Pediatric Hematology/Oncology, ISSN 1077-4114, 03/2019, Volume 41, Issue 2, pp. E72 - E78
Background: Recombinant activated factor VII (rFVIIa) has been used off-label to treat or prevent severe bleeding in patients for whom conventional treatments... 
prothrombin time | off label | bleeding | rFVIIa | pediatric hematology/oncology | MECHANISM | HEMORRHAGE | HOSPITALS | BLEEDING COMPLICATIONS | HEMOPHILIA | ONCOLOGY | THROMBOCYTOPENIA | PEDIATRICS | EXPERIENCE | INHIBITORS | HEMATOLOGY | CRITICALLY-ILL PATIENTS
Journal Article
Journal Article
Pediatric critical care medicine : a journal of the Society of Critical Care Medicine and the World Federation of Pediatric Intensive and Critical Care Societies, ISSN 1529-7535, 09/2018, Volume 19, Issue 9, pp. e472 - e478
Objectives: Pediatric intensivists frequently prescribe platelet transfusions to critically ill children, but there are limited data on platelet transfusion... 
MORTALITY | SCORE | MANAGEMENT | thrombocytopenia | THROMBOSIS | PEDIATRIC INTENSIVE-CARE | PELOD-2 | pediatric intensive care unit | transfusion-related adverse events | PEDIATRICS | extracorporeal membrane oxygenation | platelet transfusions | thromboelastography | CRITICAL CARE MEDICINE | Practice | Pediatric intensive care | Transfusion | Blood platelets | Patient outcomes
Journal Article
Pediatric Blood & Cancer, ISSN 1545-5009, 09/2014, Volume 61, Issue 9, pp. 1544 - 1550
Journal Article
Annals of the American Thoracic Society, ISSN 2325-6621, 07/2015, Volume 12, Issue 7, pp. 1097 - 1099
A 13-year-old girl with sickle cell disease secondary to sickle-cell hemoglobin presented to our institution with a chief complaint of acute chest pain and... 
RESPIRATORY SYSTEM | Bronchoscopy | Radiography | Acute Chest Syndrome - diagnosis | Bronchitis - therapy | Anemia, Sickle Cell - complications | Humans | Adolescent | Female | Lung - diagnostic imaging | Pulmonary Atelectasis - therapy | Bronchitis - diagnosis
Journal Article
Journal Article