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A phase 1 dose-escalation study: safety, tolerability, and pharmacokinetics of FBS0701, a novel oral iron chelator for the treatment of transfusional iron overload.

artículo científico publicado en 2010

A pilot study of subcutaneous decitabine in β-thalassemia intermedia ⬇️

artículo científico publicado el 23 de junio de 2011

Acute silent cerebral ischemic events in children with sickle cell anemia.

artículo científico publicado en 2013

American Society of Hematology 2020 guidelines for sickle cell disease: transfusion support

scientific article published on 01 January 2020

Beliefs about chelation among thalassemia patients

artículo científico publicado en 2012

Beta Thalassemia: Monitoring and New Treatment Approaches

artículo científico publicado en 2019

Bone disease in thalassemia: a frequent and still unresolved problem

artículo científico publicado en 2009

Central Nervous System Events in Children with Sickle Cell Disease Presenting Acutely with Headache ⬇️

artículo científico publicado el 25 de marzo de 2011

Characterization of low bone mass in young patients with thalassemia by DXA, pQCT and markers of bone turnover ⬇️

artículo científico publicado el 5 de abril de 2011

Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency

artículo científico publicado en 2020

Chelation use and iron burden in North American and British thalassemia patients: a report from the Thalassemia Longitudinal Cohort ⬇️

artículo científico publicado el 25 de enero de 2012

Chronic transfusion practice for children with sickle cell anaemia and stroke

artículo científico publicado en 2008

Combination Oral Chelation in Adult Patients With Transfusion-dependent Thalassemia and High Iron Burden

artículo científico publicado en 2019

Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia

artículo científico publicado en 2014

Corrigendum: The Spectrum of SPTA1-Associated Hereditary Spherocytosis

scientific article published on 18 October 2019

Cranial epidural hematomas: A case series and literature review of this rare complication associated with sickle cell disease

artículo científico publicado en 2016

Current recommendations for chelation for transfusion-dependent thalassemia

artículo científico publicado en 2016

Deferiprone for the treatment of transfusional iron overload in thalassemia

artículo científico publicado en 2017

Differences in the prevalence of growth, endocrine and vitamin D abnormalities among the various thalassaemia syndromes in North America

artículo científico publicado en 2009

Dysregulated arginine metabolism and cardiopulmonary dysfunction in patients with thalassaemia

artículo científico publicado en 2015

Education and employment status of children and adults with thalassemia in North America

artículo científico publicado en 2010

Effect of transfusion therapy on transcranial doppler ultrasonography velocities in children with sickle cell disease ⬇️

artículo científico publicado el 23 de diciembre de 2010

Effects of hydroxyurea treatment for patients with hemoglobin SC disease

artículo científico publicado en 2015

Elevated blood flow velocity in the anterior cerebral artery and stroke risk in sickle cell disease: extended analysis from the STOP trial.

artículo científico publicado en 2006

Epidemiologic and clinical characteristics of nontransfusion-dependent thalassemia in the United States.

artículo científico publicado en 2018

Epidemiology of human parvovirus B19 in children with sickle cell disease ⬇️

artículo científico publicado el 2 de octubre de 2003

Evaluation and treatment of transfusional iron overload in children

artículo científico

Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy ⬇️

artículo científico publicado en 2019

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

article

Gene therapy of hemoglobinopathies: progress and future challenges

artículo científico publicado en 2019

Genotype-phenotype correlation and molecular heterogeneity in pyruvate kinase deficiency

artículo científico publicado en 2020

Guidelines for the Standard Monitoring of Patients With Thalassemia: Report of the Thalassemia Longitudinal Cohort

artículo científico publicado en 2015

Headache and migraine in children with sickle cell disease are associated with lower hemoglobin and higher pain event rates but not silent cerebral infarction.

artículo científico publicado en 2014

Headache in children with sickle cell disease: prevalence and associated factors

artículo científico publicado en 2007

Hemoglobin H-constant spring in North America: an alpha thalassemia with frequent complications

artículo científico publicado en 2009

Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia-TCD With Transfusions Changing to Hydroxyurea (TWiTCH): a multicentre, open-label, phase 3, non-inferiority tria

artículo científico publicado en 2015

Inadequate dietary intake in patients with thalassemia

artículo científico publicado en 2012

Increased leucocyte apoptosis in transfused β-thalassaemia patients

artículo científico publicado en 2012

Increased prevalence of potential right-to-left shunting in children with sickle cell anaemia and stroke

artículo científico publicado en 2016

Increasing prevalence of thalassemia in America: Implications for primary care

artículo científico publicado en 2015

Inflammation and oxidant-stress in beta-thalassemia patients treated with iron chelators deferasirox (ICL670) or deferoxamine: an ancillary study of the Novartis CICL670A0107 trial

artículo científico publicado en 2008

Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease ⬇️

artículo científico publicado en 2016

Interim Results from the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy (LentiGlobin) for the Treatment of Transfusion-Dependent β-Thalassemia ⬇️

scholarly article

Intracranial aneurysms in sickle cell anemia: clinical and imaging findings

artículo científico

Iron chelation adherence to deferoxamine and deferasirox in thalassemia

artículo científico publicado en 2011

Iron chelation therapy in sickle-cell disease and other transfusion-dependent anemias

artículo científico publicado en 2004

Ischemic stroke in children and young adults with sickle cell disease in the post-STOP era

artículo científico publicado en 2019

Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy (GT): Updated Results in Group C Patients from the Phase 1/2 Hgb-206 Study ⬇️

artículo científico publicado en 2020

Liver iron concentration measurements by MRI in chronically transfused children with sickle cell anemia: baseline results from the TWiTCH trial

artículo científico publicado en 2015

Long-Term Safety and Efficacy of Mitapivat (AG-348), a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency: The DRIVE PK Study ⬇️

scholarly article

Management of transfusional iron overload – differential properties and efficacy of iron chelating agents ⬇️

artículo científico publicado el 21 de septiembre de 2011

Matched Sibling Donor Hematopoietic Stem Cell Transplantation to Prevent Stroke in Children With Sickle Cell Anemia

scientific article published on 01 January 2019

Neutropenia following IVIG therapy in pediatric patients with immune-mediated thrombocytopenia

artículo científico publicado en 2005

Oral iron chelators

artículo científico publicado en 2008

Oral iron chelators

artículo científico publicado en 2010

Pain as an emergent issue in thalassemia

artículo científico publicado en 2010

Population analysis of the alpha hemoglobin stabilizing protein (AHSP) gene identifies sequence variants that alter expression and function

article

Pregnancy outcomes in women with thalassemia in North America and the United Kingdom

artículo científico publicado en 2013

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia ⬇️

scholarly article

Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate Kinase Deficiency Natural History Study

artículo científico publicado en 2018

Prevalence of intracranial stenosis and silent cerebral infarcts in children with sickle cell anemia and low risk of stroke

artículo científico publicado en 2013

Prolongation of the prothrombin time and activated partial thromboplastin time in children with sickle cell disease

artículo científico publicado en 2006

Quality of life in thalassemia: A comparison of SF‐36 results from the thalassemia longitudinal cohort to reported literature and the US norms ⬇️

artículo científico publicado el 1 de enero de 2011

Real-World Use of Iron Chelators ⬇️

artículo científico publicado el 1 de enero de 2011

Red cell alloimmunization in a diverse population of transfused patients with thalassaemia ⬇️

artículo científico publicado el 17 de febrero de 2011

Relationship between chronic transfusion therapy and body composition in subjects with thalassemia

artículo científico publicado en 2010

Relative response of patients with myelodysplastic syndromes and other transfusion-dependent anaemias to deferasirox (ICL670): a 1-yr prospective study

artículo científico publicado en 2007

Renal dysfunction in patients with thalassaemia ⬇️

artículo científico publicado el 21 de febrero de 2011

Risk factors and mortality associated with an elevated tricuspid regurgitant jet velocity measured by Doppler-echocardiography in thalassemia: a Thalassemia Clinical Research Network report ⬇️

artículo científico publicado el 19 de julio de 2011

Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency

scientific article published on 01 September 2019

Safety and Feasibility of Hematopoietic Progenitor Stem Cell Collection by Mobilization with Plerixafor Followed by Apheresis vs Bone Marrow Harvest in Patients with Sickle Cell Disease in the Multi-center HGB-206 Trial

scientific article published on 13 May 2020

Safety and efficacy of pegylated interferon alpha-2a and ribavirin for the treatment of hepatitis C in patients with thalassemia

artículo científico publicado en 2008

Safety and pharmacokinetics of the oral iron chelator SP-420 in β-thalassemia.

artículo científico publicado en 2017

Safety of deep sedation in young children with sickle cell disease: a retrospective cohort study

artículo científico publicado en 2015

Schwann cell-conditioned medium inhibits angiogenesis in vitro and in vivo

artículo científico publicado en 2000

Serum ferritin level changes in children with sickle cell disease on chronic blood transfusion are nonlinear and are associated with iron load and liver injury

artículo científico publicado en 2009

Sickle cell anemia: intracranial stenosis and silent cerebral infarcts in children with low risk of stroke

artículo científico publicado en 2014

Sickle cell disease and transcranial Doppler imaging: inter-hemispheric differences in blood flow Doppler parameters

artículo científico publicado en 2010

Sickle cell disease: ratio of blood flow velocity of intracranial to extracranial cerebral arteries--initial experience

artículo científico publicado en 2009

Sildenafil therapy in thalassemia patients with Doppler-defined risk of pulmonary hypertension

artículo científico publicado en 2013

Silent infarcts in young children with sickle cell disease

scientific article published on 04 June 2009

Stroke and elevated blood flow velocity in the anterior cerebral artery in sickle cell disease

scientific article published on 01 May 2004

Stroke in patients with sickle cell disease

artículo científico publicado en 2013

Symptoms of depression and anxiety in patients with thalassemia: prevalence and correlates in the thalassemia longitudinal cohort.

artículo científico publicado en 2010

The Spectrum of SPTA1-Associated Hereditary Spherocytosis ⬇️

artículo científico publicado en 2019

The clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study.

artículo científico publicado en 2018

The phenotypic spectrum of germline variants: from isolated sideroblastic anemia to mitochondrial myopathy, lactic acidosis and sideroblastic anemia 2

artículo científico publicado en 2018

The pyruvate kinase (PK) to hexokinase enzyme activity ratio and erythrocyte PK protein level in the diagnosis and phenotype of PK deficiency

artículo científico publicado en 2020

Therapeutic phlebotomy is safe in children with sickle cell anaemia and can be effective treatment for transfusional iron overload

artículo científico publicado en 2015

Therapy induced iron deficiency in children treated with eltrombopag for immune thrombocytopenia.

artículo científico publicado en 2017

Transcranial Doppler ultrasonography and prophylactic transfusion program is effective in preventing overt stroke in children with sickle cell disease

artículo científico publicado en 2010

Transcranial Doppler ultrasonography in infants with sickle cell disease-Is earlier identification of children at risk of stroke possible?

scientific article published on 01 February 2010

Transcranial Doppler ultrasonography in siblings with sickle cell disease

scientific article published on 01 June 2003

Transcranial doppler re-screening of subjects who participated in STOP and STOP II.

artículo científico publicado en 2016

Transfusion and chelation practices in sickle cell disease: a regional perspective

artículo científico publicado en 2010

Transfusional Iron Overload in a Cohort of Children with Sickle Cell Disease: Impact of Magnetic Resonance Imaging, Transfusion Method, and Chelation

artículo científico publicado en 2016

Transfusional iron overload in children with sickle cell anemia on chronic transfusion therapy for secondary stroke prevention ⬇️

artículo científico publicado el 25 de noviembre de 2011

Unrelated Donor Transplantation in Children with Thalassemia Using Reduced Intensity Conditioning - the URTH Trial.

artículo científico publicado en 2018

Validation and reliability of a disease-specific quality of life measure (the TranQol) in adults and children with thalassaemia major

artículo científico publicado en 2013

Variance of pain prevalence and associated severity during the transfusion cycle of adult thalassaemia patients

artículo científico publicado en 2014

Zinc supplementation improves bone density in patients with thalassemia: a double-blind, randomized, placebo-controlled trial

artículo científico publicado en 2013

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