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Transformative effect of a Humanitarian Program for individuals affected by rare diseases: building support systems and creating local expertise

Orphanet journal of rare diseases, 2022-04, Vol.17 (1), p.87-87, Article 87

2022

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Ineffective Erythropoiesis in β-Thalassemia

TheScientificWorld, 2013-01, Vol.2013 (1), p.394295-394295

2013

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Sickle Cell Disease: New Opportunities and Challenges in Africa

TheScientificWorld, 2013-01, Vol.2013 (1), p.193252-193252

2013

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Splenectomy and thrombosis: the case of thalassemia intermedia

Journal of thrombosis and haemostasis, 2010-10, Vol.8 (10), p.2152-2158

2010

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5613049 THE DIAGNOSTIC ACCURACY AND REPEATABILITY OF AN ARTIFICIAL INTELLIGENCE BASED SYSTEM TO OBTAIN AUTOMATED LIVER IRON CONCENTRATION MEASUREMENTS USING MAGNETIC RESONANCE IMAGING

HemaSphere, 2023-04, Vol.7 (S1), p.3-4

2023

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Jaw bones’ involvement and dental features of type I and type III Gaucher disease: a radiographic study of 42 paediatric patients

European archives of paediatric dentistry, 2020-04, Vol.21 (2), p.241-247

2020

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P1505: USING ARTIFICIAL INTELLIGENCE NEURAL NETWORKS TO OBTAIN AUTOMATED LIVER IRON CONCENTRATION MEASUREMENTS USING MAGNETIC RESONANCE IMAGING – A MULTI‐SCANNER VALIDATION STUDY

HemaSphere, 2022-06, Vol.6 (Suppl), p.1386-1387

2022

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The Impact of Migrations on the Health Services for Rare Diseases in Europe: The Example of Haemoglobin Disorders

TheScientificWorld, 2013-01, Vol.2013 (1), p.727905-727905

2013

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Prenatal diagnosis for thalassaemia in Egypt: what changed parents' attitude?

Prenatal diagnosis, 2012-08, Vol.32 (8), p.777-782

2012

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Efficacy and safety of deferasirox at low and high iron burdens: results from the EPIC magnetic resonance imaging substudy

Annals of hematology, 2013-01, Vol.92 (2), p.211-219

2013

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A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease

The New England journal of medicine, 2019-08, Vol.381 (6), p.509-519

2019

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Prediction of factor VIII inhibitor development in the SIPPET cohort by mutational analysis and factor VIII antigen measurement

Journal of thrombosis and haemostasis, 2018-04, Vol.16 (4), p.778-790

2018

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Phytomedicines and Nutraceuticals: Alternative Therapeutics for Sickle Cell Anemia

TheScientificWorld, 2013-01, Vol.2013 (1), p.269659-269659

2013

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Enzyme replacement therapy and bony changes in Egyptian paediatric Gaucher disease patients

Journal of inherited metabolic disease, 2006-02, Vol.29 (1), p.92-98

2006

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A 1-year randomized controlled trial of deferasirox vs deferoxamine for myocardial iron removal in β-thalassemia major (CORDELIA)

Blood, 2014-03, Vol.123 (10), p.1447-1454

2014

Volltextzugriff (PDF)

S115: LONG-TERM EFFICACY AND SAFETY OF DEFERIPRONE FOR PATIENTS WITH SICKLE CELL DISEASE OR OTHER ANEMIAS

HemaSphere, 2022-01, Vol.6 (Suppl), p.8-8

2022

Volltextzugriff (PDF)

LONG-TERM SAFETY AND EFFICACY OF DEFERIPRONE FOR THE TREATMENT OF CHRONICALLY TRANSFUSED, IRON-OVERLOADED PATIENTS WITH SICKLE CELL DISEASE OR OTHER ANEMIAS

Hematology, Transfusion and Cell Therapy, 2021-10, Vol.43, p.S23-S24

2021

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Genetic risk stratification to reduce inhibitor development in the early treatment of hemophilia A: a SIPPET analysis

Blood, 2017-10, Vol.130 (15), p.1757-1759

2017

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Nonneutralizing antibodies against factor VIII and risk of inhibitor development in severe hemophilia A

Blood, 2017-03, Vol.129 (10), p.1245-1250

2017

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International survey of T2 cardiovascular magnetic resonance in β-thalassemia major

Haematologica (Roma), 2013-09, Vol.98 (9), p.1368-1374

2013

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Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes: Results from the large 1-year EPIC study

Leukemia research, 2010-09, Vol.34 (9), p.1143-1150

2010

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Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias

Haematologica (Roma), 2010-04, Vol.95 (4), p.557-566

2010

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Iron chelation therapy with deferasirox in patients with aplastic anemia: a subgroup analysis of 116 patients from the EPIC trial

Blood, 2010-10, Vol.116 (14), p.2448-2454

2010

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The use of DNA markers for carrier detection and prenatal diagnosis of haemophilia A in Egyptian families

Haemophilia : the official journal of the World Federation of Hemophilia, 2008-09, Vol.14 (5), p.1082-1087

2008

Volltextzugriff (PDF)

S123: SAFETY AND EFFICACY OF EARLY-START DEFERIPRONE IN INFANTS AND YOUNG CHILDREN WITH BETA-THALASSEMIA (START STUDY)

HemaSphere, 2022-01, Vol.6 (Suppl), p.12-13

2022

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Transformative effect of a Humanitarian Program for individuals affected by rare diseases: building support systems and creating local expertise

Ineffective Erythropoiesis in β-Thalassemia

Sickle Cell Disease: New Opportunities and Challenges in Africa

Splenectomy and thrombosis: the case of thalassemia intermedia

5613049 THE DIAGNOSTIC ACCURACY AND REPEATABILITY OF AN ARTIFICIAL INTELLIGENCE BASED SYSTEM TO OBTAIN AUTOMATED LIVER IRON CONCENTRATION MEASUREMENTS USING MAGNETIC RESONANCE IMAGING

Jaw bones’ involvement and dental features of type I and type III Gaucher disease: a radiographic study of 42 paediatric patients

P1505: USING ARTIFICIAL INTELLIGENCE NEURAL NETWORKS TO OBTAIN AUTOMATED LIVER IRON CONCENTRATION MEASUREMENTS USING MAGNETIC RESONANCE IMAGING – A MULTI‐SCANNER VALIDATION STUDY

The Impact of Migrations on the Health Services for Rare Diseases in Europe: The Example of Haemoglobin Disorders

Prenatal diagnosis for thalassaemia in Egypt: what changed parents' attitude?

Efficacy and safety of deferasirox at low and high iron burdens: results from the EPIC magnetic resonance imaging substudy

A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease

Prediction of factor VIII inhibitor development in the SIPPET cohort by mutational analysis and factor VIII antigen measurement

Phytomedicines and Nutraceuticals: Alternative Therapeutics for Sickle Cell Anemia

Enzyme replacement therapy and bony changes in Egyptian paediatric Gaucher disease patients

A 1-year randomized controlled trial of deferasirox vs deferoxamine for myocardial iron removal in β-thalassemia major (CORDELIA)

S115: LONG-TERM EFFICACY AND SAFETY OF DEFERIPRONE FOR PATIENTS WITH SICKLE CELL DISEASE OR OTHER ANEMIAS

LONG-TERM SAFETY AND EFFICACY OF DEFERIPRONE FOR THE TREATMENT OF CHRONICALLY TRANSFUSED, IRON-OVERLOADED PATIENTS WITH SICKLE CELL DISEASE OR OTHER ANEMIAS

Genetic risk stratification to reduce inhibitor development in the early treatment of hemophilia A: a SIPPET analysis

Nonneutralizing antibodies against factor VIII and risk of inhibitor development in severe hemophilia A

International survey of T2 cardiovascular magnetic resonance in β-thalassemia major

Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes: Results from the large 1-year EPIC study

Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias

Iron chelation therapy with deferasirox in patients with aplastic anemia: a subgroup analysis of 116 patients from the EPIC trial

The use of DNA markers for carrier detection and prenatal diagnosis of haemophilia A in Egyptian families

S123: SAFETY AND EFFICACY OF EARLY-START DEFERIPRONE IN INFANTS AND YOUNG CHILDREN WITH BETA-THALASSEMIA (START STUDY)

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