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Pre-transplantation iron chelation in patients with MDS or acute leukemia and iron overload undergoing myeloablative allo-SCT

Bone marrow transplantation (Basingstoke), 2013-01, Vol.48 (1), p.146-147

2013

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

American journal of hematology, 2011-01, Vol.86 (1), p.92-95

2011

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Deletion of SLC19A2, the high affinity thiamine transporter, causes selective inner hair cell loss and an auditory neuropathy phenotype

Journal of the Association for Research in Otolaryngology, 2006-09, Vol.7 (3), p.211-217

2006

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The gene mutated in thiamine-responsive anaemia with diabetes and deafness (TRMA) encodes a functional thiamine transporter

Nature genetics, 1999-07, Vol.22 (3), p.305-308

1999

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Transition considerations for extended half-life factor products

Haemophilia : the official journal of the World Federation of Hemophilia, 2015-05, Vol.21 (3), p.285-288

2015

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Recognizing the need for personalization of haemophilia patient-reported outcomes in the prophylaxis era

Haemophilia : the official journal of the World Federation of Hemophilia, 2016-11, Vol.22 (6), p.825-832

2016

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Combined schedule of 7-valent pneumococcal conjugate vaccine followed by 23-valent pneumococcal vaccine in children and young adults with sickle cell disease

The Journal of pediatrics, 1998-08, Vol.133 (2), p.275-278

1998

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Immunogenicity, efficacy and safety of Nuwiq® (human‐cl rhFVIII) in previously untreated patients with severe haemophilia A—Interim results from the NuProtect Study

Haemophilia : the official journal of the World Federation of Hemophilia, 2018-03, Vol.24 (2), p.211-220

2018

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Author's response: 'Transition considerations for extended half-life factor products'

Haemophilia : the official journal of the World Federation of Hemophilia, 2015-09, Vol.21 (5), p.e454-e455

2015

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Rituximab for adolescents with haemophilia and high titre inhibitors

Haemophilia : the official journal of the World Federation of Hemophilia, 2006-05, Vol.12 (3), p.218-222

2006

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The use of a single von Willebrand factor‐containing, plasma‐derived FVIII product in hemophilia A immune tolerance induction: the US experience

Journal of thrombosis and haemostasis, 2011-11, Vol.9 (11), p.2229-2234

2011

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Recombinant porcine factor VIII for high‐risk surgery in paediatric congenital haemophilia A with high‐titre inhibitor

Haemophilia : the official journal of the World Federation of Hemophilia, 2017-03, Vol.23 (2), p.e93-e98

2017

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Correlation between dispensed and prescribed doses of factor products for bleeding disorders: can a small, centre-based pharmacy hit the mark?

Haemophilia : the official journal of the World Federation of Hemophilia, 2015-03, Vol.21 (2), p.190-195

2015

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Perioperative management of haemophilia B: A critical appraisal of the evidence and current practices

Haemophilia : the official journal of the World Federation of Hemophilia, 2017-11, Vol.23 (6), p.821-831

2017

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Coagulation abnormalities in pediatric and adult patients after sclerotherapy or embolization of vascular anomalies

American journal of roentgenology (1976), 2001-12, Vol.177 (6), p.1359

2001

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Recombinant activated factor VII in approved indications: Update on safety

Haemophilia : the official journal of the World Federation of Hemophilia, 2018-07, Vol.24 (4), p.e275-e277

2018

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Gene Therapy and Hemophilia: Where Do We Go from Here?

Journal of blood medicine, 2022-01, Vol.13, p.559-580

2022

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Mechanism of thiamine uptake by human colonocytes: studies with cultured colonic epithelial cell line NCM460

American journal of physiology: Gastrointestinal and liver physiology, 2001-07, Vol.281 (1), p.G144-G150

2001

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Asparaginase-associated lipid abnormalities in children with acute lymphoblastic leukemia

Blood, 1997-03, Vol.89 (6), p.1886-1895

1997

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Safety of recombinant activated factor VII (rFVIIa) in patients with congenital haemophilia with inhibitors: overall rFVIIa exposure and intervals following high (>240 μg kg−1) rFVIIa doses across clinical trials and registries

Haemophilia : the official journal of the World Federation of Hemophilia, 2014-01, Vol.20 (1), p.e23-e31

2014

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Assessment of individual dose utilization vs. physician prescribing recommendations for recombinant activated factor VII (rFVIIa) in paediatric and adult patients with congenital haemophilia and alloantibody inhibitors (CHwI): the Dosing Observational Study in Hemophilia (DOSE)

Haemophilia : the official journal of the World Federation of Hemophilia, 2013-07, Vol.19 (4), p.524-532

2013

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Localization of the Gene for Thiamine-Responsive Megaloblastic Anemia Syndrome, on the Long Arm of Chromosome 1, by Homozygosity Mapping

American journal of human genetics, 1997-12, Vol.61 (6), p.1335-1341

1997

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Passive cigarette smoking and reduced HDL cholesterol levels in children with high-risk lipid profiles

Circulation (New York, N.Y.), 1997-09, Vol.96 (5), p.1403-1407

1997

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Hemophilia. A new approach to an old disease

Hematology/oncology clinics of North America, 1998-12, Vol.12 (6), p.1315-1344

1998

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Sequence-Specific DNA Binding of Individual Cut Repeats of the Human CCAAT Displacement/Cut Homeodomain Protein

Proceedings of the National Academy of Sciences - PNAS, 1994-08, Vol.91 (16), p.7757-7761

1994

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Pre-transplantation iron chelation in patients with MDS or acute leukemia and iron overload undergoing myeloablative allo-SCT

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

Deletion of SLC19A2, the high affinity thiamine transporter, causes selective inner hair cell loss and an auditory neuropathy phenotype

The gene mutated in thiamine-responsive anaemia with diabetes and deafness (TRMA) encodes a functional thiamine transporter

Transition considerations for extended half-life factor products

Recognizing the need for personalization of haemophilia patient-reported outcomes in the prophylaxis era

Combined schedule of 7-valent pneumococcal conjugate vaccine followed by 23-valent pneumococcal vaccine in children and young adults with sickle cell disease

Immunogenicity, efficacy and safety of Nuwiq® (human‐cl rhFVIII) in previously untreated patients with severe haemophilia A—Interim results from the NuProtect Study

Author's response: 'Transition considerations for extended half-life factor products'

Rituximab for adolescents with haemophilia and high titre inhibitors

The use of a single von Willebrand factor‐containing, plasma‐derived FVIII product in hemophilia A immune tolerance induction: the US experience

Recombinant porcine factor VIII for high‐risk surgery in paediatric congenital haemophilia A with high‐titre inhibitor

Correlation between dispensed and prescribed doses of factor products for bleeding disorders: can a small, centre-based pharmacy hit the mark?

Perioperative management of haemophilia B: A critical appraisal of the evidence and current practices

Coagulation abnormalities in pediatric and adult patients after sclerotherapy or embolization of vascular anomalies

Recombinant activated factor VII in approved indications: Update on safety

Gene Therapy and Hemophilia: Where Do We Go from Here?

Mechanism of thiamine uptake by human colonocytes: studies with cultured colonic epithelial cell line NCM460

Asparaginase-associated lipid abnormalities in children with acute lymphoblastic leukemia

Safety of recombinant activated factor VII (rFVIIa) in patients with congenital haemophilia with inhibitors: overall rFVIIa exposure and intervals following high (>240 μg kg−1) rFVIIa doses across clinical trials and registries

Assessment of individual dose utilization vs. physician prescribing recommendations for recombinant activated factor VII (rFVIIa) in paediatric and adult patients with congenital haemophilia and alloantibody inhibitors (CHwI): the Dosing Observational Study in Hemophilia (DOSE)

Localization of the Gene for Thiamine-Responsive Megaloblastic Anemia Syndrome, on the Long Arm of Chromosome 1, by Homozygosity Mapping

Passive cigarette smoking and reduced HDL cholesterol levels in children with high-risk lipid profiles

Hemophilia. A new approach to an old disease

Sequence-Specific DNA Binding of Individual Cut Repeats of the Human CCAAT Displacement/Cut Homeodomain Protein

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