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Management of haemophilia A with inhibitors: A regional cross‐talk

Haemophilia : the official journal of the World Federation of Hemophilia, 2022-11, Vol.28 (6), p.950-961

2022

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Multicenter validation of spin-density projection-assisted R2-MRI for the noninvasive measurement of liver iron concentration

Magnetic resonance in medicine, 2014-06, Vol.71 (6), p.2215-2223

2014

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Deferiprone versus deferoxamine for transfusional iron overload in sickle cell disease and other anemias: Pediatric subgroup analysis of the randomized, open‐label FIRST study

Pediatric blood & cancer, 2024-01, Vol.71 (1), p.e30711-n/a

2024

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Risk of mortality from anemia and iron overload in nontransfusion‐dependent β‐thalassemia

American journal of hematology, 2022-02, Vol.97 (2), p.E78-E80

2022

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A complication risk score to evaluate clinical severity of thalassaemia syndromes

British journal of haematology, 2021-02, Vol.192 (3), p.626-633

2021

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Primary HBB gene mutation severity and long‐term outcomes in a global cohort of β‐thalassaemia

British journal of haematology, 2022-01, Vol.196 (2), p.414-423

2022

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Performance of a clinical risk prediction model for inhibitor formation in severe haemophilia A

Haemophilia : the official journal of the World Federation of Hemophilia, 2021-07, Vol.27 (4), p.e441-e449

2021

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Neuronopathic Gaucher disease: demographic and clinical features of 131 patients enrolled in the International Collaborative Gaucher Group Neurological Outcomes Subregistry

Journal of inherited metabolic disease, 2010-08, Vol.33 (4), p.339-346

2010

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Reversal of life-threatening hepatopulmonary syndrome in Gaucher disease by imiglucerase enzyme replacement therapy

Molecular genetics and metabolism reports, 2019-09, Vol.20, p.100490, Article 100490

2019

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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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Continuation of deferiprone therapy in patients with mild neutropenia may not lead to a more severe drop in neutrophil count

European journal of haematology, 2014-04, Vol.92 (4), p.337-340

2014

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The challenges of adherence and persistence with iron chelation therapy

International journal of hematology, 2011-11, Vol.94 (5), p.453-460

2011

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Ineffective Erythropoiesis in β-Thalassemia

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

2013

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Prevalence and distribution of iron overload in patients with transfusion-dependent anemias differs across geographic regions: results from the CORDELIA study

European journal of haematology, 2015-09, Vol.95 (3), p.244-253

2015

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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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Age‐related complications in treatment‐naïve patients with thalassaemia intermedia

British journal of haematology, 2010-08, Vol.150 (4), p.486-489

2010

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Long‐term safety and efficacy of deferasirox in young pediatric patients with transfusional hemosiderosis: Results from a 5year observational study (ENTRUST)

Pediatric blood & cancer, 2017-09, Vol.64 (9), p.n/a

2017

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Genotype–phenotype associations in familial Mediterranean fever: a study of 500 Egyptian pediatric patients

Clinical rheumatology, 2022-05, Vol.41 (5), p.1511-1521

2022

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Limitations of serum ferritin to predict liver iron concentration responses to deferasirox therapy in patients with transfusion‐dependent thalassaemia

European journal of haematology, 2017-03, Vol.98 (3), p.280-288

2017

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Serum ferritin level and morbidity risk in transfusion-independent patients with β-thalassemia intermedia: the ORIENT study

Haematologica (Roma), 2014-11, Vol.99 (11), p.e218

2014

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Updates in Hemoglobinopathies

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

2013

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Deferasirox for up to 3 years leads to continued improvement of myocardial T2 in patients with β-thalassemia major

Haematologica (Roma), 2012-06, Vol.97 (6), p.842-848

2012

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Revised recommendations for the management of Gaucher disease in children

European journal of pediatrics, 2013-04, Vol.172 (4), p.447-458

2013

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Bringing global hematology research to the forefront

Blood advances, 2024-07, Vol.8 (14), p.3607-3611

2024

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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

Volltextzugriff (PDF)

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Management of haemophilia A with inhibitors: A regional cross‐talk

Multicenter validation of spin-density projection-assisted R2-MRI for the noninvasive measurement of liver iron concentration

Deferiprone versus deferoxamine for transfusional iron overload in sickle cell disease and other anemias: Pediatric subgroup analysis of the randomized, open‐label FIRST study

Risk of mortality from anemia and iron overload in nontransfusion‐dependent β‐thalassemia

A complication risk score to evaluate clinical severity of thalassaemia syndromes

Primary HBB gene mutation severity and long‐term outcomes in a global cohort of β‐thalassaemia

Performance of a clinical risk prediction model for inhibitor formation in severe haemophilia A

Neuronopathic Gaucher disease: demographic and clinical features of 131 patients enrolled in the International Collaborative Gaucher Group Neurological Outcomes Subregistry

Reversal of life-threatening hepatopulmonary syndrome in Gaucher disease by imiglucerase enzyme replacement therapy

A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease

Continuation of deferiprone therapy in patients with mild neutropenia may not lead to a more severe drop in neutrophil count

The challenges of adherence and persistence with iron chelation therapy

Ineffective Erythropoiesis in β-Thalassemia

Prevalence and distribution of iron overload in patients with transfusion-dependent anemias differs across geographic regions: results from the CORDELIA study

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

Age‐related complications in treatment‐naïve patients with thalassaemia intermedia

Long‐term safety and efficacy of deferasirox in young pediatric patients with transfusional hemosiderosis: Results from a 5year observational study (ENTRUST)

Genotype–phenotype associations in familial Mediterranean fever: a study of 500 Egyptian pediatric patients

Limitations of serum ferritin to predict liver iron concentration responses to deferasirox therapy in patients with transfusion‐dependent thalassaemia

Serum ferritin level and morbidity risk in transfusion-independent patients with β-thalassemia intermedia: the ORIENT study

Updates in Hemoglobinopathies

Deferasirox for up to 3 years leads to continued improvement of myocardial T2 in patients with β-thalassemia major

Revised recommendations for the management of Gaucher disease in children

Bringing global hematology research to the forefront

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

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