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Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency

The New England journal of medicine, 2019-09, Vol.381 (10), p.933-944

2019

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Trans-acting genetic modifiers of clinical severity in heterozygous β-Thalassemia trait

Annals of hematology, 2024-11, Vol.103 (11), p.4437-4447

2024

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Comparison of Inline R2 MRI versus FerriScan for liver iron quantification in patients on chelation therapy for iron overload: preliminary results

European radiology, 2021-12, Vol.31 (12), p.9296-9305

2021

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Impact of MRI technique on clinical decision-making in patients with liver iron overload: comparison of FerriScan- versus R2-derived liver iron concentration

European radiology, 2020-04, Vol.30 (4), p.1959-1968

2020

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Impact of a transition program with navigator on loss to follow‐up, medication adherence, and appointment attendance in hemoglobinopathies

Pediatric blood & cancer, 2019-08, Vol.66 (8), p.e27781-n/a

2019

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Early Strokes Are Associated with More Global Cognitive Deficits in Adults with Sickle Cell Disease

Journal of clinical medicine, 2023-02, Vol.12 (4), p.1615

2023

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Bone mineral density in adult patients with pyruvate kinase deficiency on long-term mitapivat treatment

Haematologica (Roma), 2024-03, Vol.109 (3), p.963-967

2024

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THromboprophylaxis In Sickle Cell Disease with central venous catheters (THIS): an internal pilot randomised controlled trial protocol

BMJ open, 2024-01, Vol.14 (1), p.e079363-e079363

2024

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Luspatercept for transfusion-dependent β-thalassemia: time to get real

Therapeutic advances in hematology, 2023-01, Vol.14, p.20406207231195594-20406207231195594

2023

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Prevalence of elevated hemoglobin and hematocrit levels in patients with obstructive sleep apnea and the impact of treatment with continuous positive airway pressure: a meta-analysis

Hematology (Luxembourg), 2022-12, Vol.27 (1), p.889-901

2022

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Sickle cell cerebrovascular reactivity to a CO2 stimulus: Too little, too slow

Frontiers in physiology, 2022-08, Vol.13, p.886807-886807

2022

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Successful treatment of heparin induced thrombocytopenia (HIT) with fondaparinux

Thrombosis and haemostasis, 2005-05, Vol.93 (5), p.999-1000

2005

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Assessing Cerebrovascular Resistance in Patients With Sickle Cell Disease

Frontiers in physiology, 2022-03, Vol.13, p.847969-847969

2022

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Designing a single-arm phase 2 clinical trial of mitapivat for adult patients with erythrocyte membranopathies (SATISFY): a framework for interventional trials in rare anaemias – pilot study protocol

BMJ open, 2024-07, Vol.14 (7), p.e083691

2024

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Investigating the association between fasting insulin, erythrocytosis and HbA1c through Mendelian randomization and observational analyses

Frontiers in endocrinology (Lausanne), 2023-03, Vol.14, p.1146099-1146099

2023

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EspP, an Extracellular Serine Protease from Enterohemorrhagic E. coli, Reduces Coagulation Factor Activities, Reduces Clot Strength, and Promotes Clot Lysis

PloS one, 2016-03, Vol.11 (3), p.e0149830-e0149830

2016

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The Pyruvate Kinase Deficiency Global Longitudinal (Peak) Registry: rationale and study design

BMJ open, 2023-03, Vol.13 (3), p.e063605-e063605

2023

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Screening for Cognitive Dysfunction Using the Rowland Universal Dementia Assessment Scale in Adults With Sickle Cell Disease

JAMA network open, 2021-05, Vol.4 (5), p.e217039-e217039

2021

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Eculizumab and Beyond: The Past, Present, and Future of Complement Therapeutics

Transfusion medicine reviews, 2019-10, Vol.33 (4), p.256-265

2019

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Untreated Anemia in Nontransfusion‐dependent β‐thalassemia: Time to Sound the Alarm

HemaSphere, 2022-12, Vol.6 (12), p.e806-n/a

2022

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Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or β-thalassaemia: an open-label, multicentre, phase 2 study

The Lancet (British edition), 2022-08, Vol.400 (10351), p.493-501

2022

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Pyruvate kinase activators: targeting red cell metabolism in thalassemia

Hematology, 2023-12, Vol.2023 (1), p.114-120

Kuo, Kevin H.M.

2023

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Go the Distance: Reproductive Health Care for People with Sickle Cell Disease

Hematology/oncology clinics of North America, 2022-12, Vol.36 (6), p.1255-1270

2022

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Assessment of cerebrovascular function in patients with sickle cell disease using transfer function analysis

Physiological reports, 2022-10, Vol.10 (19), p.e15472-n/a

2022

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Prophylactic transfusion for pregnant women with sickle cell disease: a systematic review and meta-analysis

Blood, 2015-11, Vol.126 (21), p.2424-2435

2015

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Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency

Trans-acting genetic modifiers of clinical severity in heterozygous β-Thalassemia trait

Comparison of Inline R2 MRI versus FerriScan for liver iron quantification in patients on chelation therapy for iron overload: preliminary results

Impact of MRI technique on clinical decision-making in patients with liver iron overload: comparison of FerriScan- versus R2-derived liver iron concentration

Impact of a transition program with navigator on loss to follow‐up, medication adherence, and appointment attendance in hemoglobinopathies

Early Strokes Are Associated with More Global Cognitive Deficits in Adults with Sickle Cell Disease

Bone mineral density in adult patients with pyruvate kinase deficiency on long-term mitapivat treatment

THromboprophylaxis In Sickle Cell Disease with central venous catheters (THIS): an internal pilot randomised controlled trial protocol

Luspatercept for transfusion-dependent β-thalassemia: time to get real

Prevalence of elevated hemoglobin and hematocrit levels in patients with obstructive sleep apnea and the impact of treatment with continuous positive airway pressure: a meta-analysis

Sickle cell cerebrovascular reactivity to a CO2 stimulus: Too little, too slow

Successful treatment of heparin induced thrombocytopenia (HIT) with fondaparinux

Assessing Cerebrovascular Resistance in Patients With Sickle Cell Disease

Designing a single-arm phase 2 clinical trial of mitapivat for adult patients with erythrocyte membranopathies (SATISFY): a framework for interventional trials in rare anaemias – pilot study protocol

Investigating the association between fasting insulin, erythrocytosis and HbA1c through Mendelian randomization and observational analyses

EspP, an Extracellular Serine Protease from Enterohemorrhagic E. coli, Reduces Coagulation Factor Activities, Reduces Clot Strength, and Promotes Clot Lysis

The Pyruvate Kinase Deficiency Global Longitudinal (Peak) Registry: rationale and study design

Screening for Cognitive Dysfunction Using the Rowland Universal Dementia Assessment Scale in Adults With Sickle Cell Disease

Eculizumab and Beyond: The Past, Present, and Future of Complement Therapeutics

Untreated Anemia in Nontransfusion‐dependent β‐thalassemia: Time to Sound the Alarm

Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or β-thalassaemia: an open-label, multicentre, phase 2 study

Pyruvate kinase activators: targeting red cell metabolism in thalassemia

Go the Distance: Reproductive Health Care for People with Sickle Cell Disease

Assessment of cerebrovascular function in patients with sickle cell disease using transfer function analysis

Prophylactic transfusion for pregnant women with sickle cell disease: a systematic review and meta-analysis

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