J. Pasi

发表

Clinical Considerations for Capsid Choice in the Development of Liver-Targeted AAV-Based Gene Transfer

Valérie Ferreira, F. Leebeek, E. Sawyer, 2019, Molecular therapy. Methods & clinical development.

Comprehensive Cancer-Predisposition Gene Testing in an Adult Multiple Primary Tumor Series Shows a Broad Range of Deleterious Variants and Atypical Tumor Phenotypes

William J. Astle, Dorothy A. Thompson, Sri V. V. Deevi, 2018, American journal of human genetics.

Identification of type 1 von Willebrand disease patients with reduced von Willebrand factor survival by assay of the VWF propeptide in the European study: molecular and clinical markers for the diagnosis and management of type 1 VWD (MCMDM-1VWD).

J. Goudemand, S. Lethagen, J. Eikenboom, 2008, Blood.

Detailed von Willebrand factor multimer analysis in patients with von Willebrand disease in the European study, molecular and clinical markers for the diagnosis and management of type 1 von Willebrand disease (MCMDM‐1VWD)

J. Goudemand, S. Lethagen, J. Eikenboom, 2008, Journal of thrombosis and haemostasis : JTH.

Phenotype and genotype of a cohort of families historically diagnosed with type 1 von Willebrand disease in the European study, Molecular and Clinical Markers for the Diagnosis and Management of Type 1 von Willebrand Disease (MCMDM-1VWD).

J. Goudemand, C. Halldén, S. Lethagen, 2007, Blood.

Linkage analysis in families diagnosed with type 1 von Willebrand disease in the European study, molecular and clinical markers for the diagnosis and management of type 1 VWD

J. Goudemand, S. Lethagen, J. Eikenboom, 2006, Journal of thrombosis and haemostasis : JTH.

The effects of verapamil, diltiazem, nifedipine and propranolol on metabolic control in hypertensives with non-insulin dependent diabetes mellitus.

B. Singh, M. Chellingsworth, J. Pasi, 1989, Journal of human hypertension.

Role of the Thrombelastograph as an adjunctive test in thrombophilia screening

D. Perry, G. Hamilton, A. Handa, 2004, Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis.

Epidemiology and pathophysiology of cancer-associated thrombosis

S. Noble, J. Pasi, 2010, British Journal of Cancer.

Beyond patient benefit: clinical development in hemophilia

G. Auerswald, G. Benson, T. Lambert, 2012, Hematology.

Experiences with continuous infusion of recombinant activated factor VII.

M. Morfini, J. Goudemand, C. Négrier, 1998, Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis.

Novel manifestations of immune dysregulation and granule defects in gray platelet syndrome

William J. Astle, Sri V. V. Deevi, Matthew C. Sims, 2020, medRxiv.

SY1. building the foundation for investigator-initiated clinical trial

P. Jänne, H. Katayama, M. Kudo, 2012 .

Psychological interventions for people with hemophilia.

A. Iorio, S. Nevitt, J. Pasi, 2012, The Cochrane database of systematic reviews.

Evaluation of thromboelastography for monitoring recombinant activated factor VII ex vivo in haemophilia A and B patients with inhibitors: a multicentre trial

C. Négrier, B. Konkle, D. Viuff, 2008, Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis.

Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A

J. Mahlangu, R. Liesner, B. Nolan, 2015, Journal of thrombosis and haemostasis : JTH.

Novel manifestations of immune dysregulation and granule defects in gray platelet syndrome

William J. Astle, Sri V. V. Deevi, Matthew C. Sims, 2020, medRxiv.

Diagnosis and management of von Willebrand disease in the United Kingdom.

P. Collins, A. Cumming, S. Keeney, 2011, Seminars in thrombosis and hemostasis.

Correction of both prothrombin time and primary haemostasis by recombinant factor VII during therapeutic alcohol injection of hepatocellular cancer in liver cirrhosis.

A. Burroughs, S. Chung, J. Pasi, 1999, Journal of hepatology.

Response to desmopressin is influenced by the genotype and phenotype in type 1 von Willebrand disease (VWD): results from the European Study MCMDM-1VWD.

A. Tosetto, J. Goudemand, S. Lethagen, 2008, Blood.

Von Willebrand Factor Modulates Factor VIII Immunogenicity: Comparative Study of Different Factor VIII Concentrates in a Haemophilia A Mouse Model

J. Saint-Remy, R. Kotitschke, J. Pasi, 2002, Thrombosis and Haemostasis.

A ploidy-agnostic method for estimating telomere length from whole genome sequencing data.

Ania, Eleanor, Dewhurst, 2018 .

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

D. Perry, M. Laffan, S. Rangarajan, 2017, The New England journal of medicine.

Membranous obstruction of the inferior vena cava in a patient with factor V Leiden: evidence for a post-thrombotic aetiology.

J. Pasi, A. Burroughs, R. Dick, 1997, Journal of hepatology.

Performance of the platelet function analyser PFA-100 in testing abnormalities of primary haemostasis.

R. Liesner, R. Liesner, I. Mackie, 1999, Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis.

Reply to the letter to the editor 'clinical benefit of continuing ALK inhibition with crizotinib beyond initial disease progression in patients with advanced ALK-positive NSCLC' by Ou et al.

S. Ou, J. Pasi, 2014, Annals of oncology : official journal of the European Society for Medical Oncology.

Adenovirus‐associated antibodies in UK cohort of hemophilia patients: A seroprevalence study of the presence of adenovirus‐associated virus vector–serotypes AAV5 and AAV8 neutralizing activity and antibodies in patients with hemophilia A

D. Perry, S. Fong, S. Rangarajan, 2019, Research and practice in thrombosis and haemostasis.

Efficacy and safety of simoctocog alfa (Nuwiq®) in patients with severe hemophilia A: a review of clinical trial data from the GENA program

J. Oldenburg, C. Kessler, R. Klamroth, 2019, Therapeutic advances in hematology.

Pharmacokinetic In Vivo Comparison Using 1-Stage and Chromogenic Substrate Assays with Two Formulations of Hemofil-M

E. Gomperts, C. Lee, G. Bray, 1996, Thrombosis and Haemostasis.

An investigation of the von Willebrand factor genotype in UK patients diagnosed to have type 1 von Willebrand disease

P. Collins, C. Hay, D. Keeling, 2006, Thrombosis and Haemostasis.

Wilate use in 47 children with von Willebrand disease: the North London paediatric haemophilia network experience

K. Khair, Y.-H. Chen, R. Liesner, 2015, Haemophilia : the official journal of the World Federation of Hemophilia.

FEIBA prophylaxis in haemophilia patients: a clinical update and treatment recommendations

D. Perry, G. Dolan, M. Laffan, 2010, Haemophilia : the official journal of the World Federation of Hemophilia.

Factor IX inhibitors and anaphylaxis in hemophilia B.

J. Katz, R. Ljung, N. Key, 1997, Journal of pediatric hematology/oncology.

Switching to recombinant factor IX Fc fusion protein prophylaxis results in fewer infusions, decreased factor IX consumption and lower bleeding rates

M. Ozelo, C. Négrier, A. Shapiro, 2015, British journal of haematology.

Humanistic burden of problem joints for children and adults with haemophilia

K. Khair, M. Laffan, J. O’Hara, 2022, Haemophilia : the official journal of the World Federation of Hemophilia.

The impact of factor infusion frequency on health-related quality of life in people with haemophilia

K. Khair, J. O’Hara, J. Pasi, 2020 .

Factor IX inhibitors and anaphylaxis in hemophilia B.

J. Katz, R. Ljung, N. Key, 1997, Haemophilia : the official journal of the World Federation of Hemophilia.

Long-Term Safety and Efficacy of Recombinant Factor VIII FC (RFVIIIFC) in Adults and Adolescents With Severe Haemophilia A: An Interim Analysis of The ASPIRE Study

D. Perry, B. Konkle, J. Mahlangu, 2015 .

Long‐acting recombinant factor IX Fc fusion protein (rFIXFc) for perioperative management of subjects with haemophilia B in the phase 3 B‐LONG study

C. Hermans, D. Perry, M. Ozelo, 2015, British journal of haematology.

Factor IX inhibitors and anaphylaxis in haemophilia B

N. Key, I. Warrier, G. Rivard, 1996, Haemophilia : the official journal of the World Federation of Hemophilia.

Treatment of bleeding episodes with recombinant factor VIII Fc fusion protein in A‐LONG study subjects with severe haemophilia A

D. Perry, D. Quon, J. Mahlangu, 2017, Haemophilia : the official journal of the World Federation of Hemophilia.

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

Pratima Chowdary, Savita Rangarajan, Cecilia Rosales, 2011, The New England journal of medicine.

A quantitative analysis of bleeding symptoms in type 1 von Willebrand disease: results from a multicenter European study (MCMDM‐1 VWD)

A. Tosetto, J. Goudemand, S. Lethagen, 2006, Journal of thrombosis and haemostasis : JTH.

Hypercoagulability in patients with primary biliary cirrhosis and primary sclerosing cholangitis evaluated by thrombelastography.

A. Burroughs, D. Patch, J. Pasi, 1997, Journal of hepatology.

The first Team Haemophilia Education meeting, 2015, Amsterdam, The Netherlands

P. Giangrande, D. Perry, A. Miners, 2016, European journal of haematology.

Improvement in pain-related quality of life in patients with hemophilia A treated with rFVIIIFc individualized prophylaxis: post hoc analysis from the A-LONG study

C. Hermans, S. Aballéa, J. Pasi, 2022, Therapeutic advances in hematology.

European curriculum for thrombosis and haemostasis

J. Astermark, C. Hermans, C. Négrier, 2009, Haemophilia : the official journal of the World Federation of Hemophilia.

First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

J. Astermark, C. Hermans, Michael Wang, 2020 .

Safety, Efficacy, and Pharmacokinetics of Recombinant Factor VIII FC Fusion Protein (RFVIIIFC) in Previously Treated Paediatric Subjects with Severe Haemophilia A (Kids A-LONG)

J. Mahlangu, B. Nolan, G. Young, 2013 .

Natural history and clinical characteristics of inhibitors in previously treated haemophilia A patients: a case series

A. Iorio, T. Lambert, M. Makris, 2017, Haemophilia : the official journal of the World Federation of Hemophilia.

Interim Analysis Of The ASPIRE Study Evaluating Long-term Safety And Efficacy Of Recombinant Factor VIII FC (RFVIIIFC) In Children With Severe Haemophilia A

J. Mahlangu, B. Nolan, G. Young, 2015 .

Subject-Reported Changes in Physical Activity During The B-LONG Study of Recombinant Factor IX FC Fusion Protein (RFIXFC) For Severe Haemophilia B

M. Ozelo, M. Ragni, J. Windyga, 2015 .

Safety, Efficacy, and Pharmacokinetics of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously-Treated Children with Severe Hemophilia a (Kids-ALONG)

J. Mahlangu, B. Nolan, G. Young, 2014 .

Somatic gene therapy for haemophilia: Some counselling issues for today and tomorrow

R. Miller, D. Perry, P. Fields, 2002 .

Impact of plasma von Willebrand factor levels in the diagnosis of type 1 von Willebrand disease: results from a multicenter European study (MCMDM‐1VWD)

A. Tosetto, J. Goudemand, S. Lethagen, 2007, Journal of thrombosis and haemostasis : JTH.

A rare cause of neonatal hydrocephalus

S. Kempley, J. Pasi, S. Davidson, 2013 .

Morbidity and mortality from transfusion- transmitted disease in haemophilia

C. Sabin, Christine A. Lee, A. Phillips, 1995, The Lancet.

De Novo Truncating Mutations in WASF1 Cause Intellectual Disability with Seizures

Henning Gall, Fengyuan Hu, Kevin Rue-Albrecht, 2018, American journal of human genetics.

Propelling Healthcare with Advanced Therapy Medicinal Products: A Policy Discussion

A. Metspalu, D. Toncheva, C. Hermans, 2020, Biomedicine Hub.

An unusual cause of bleeding in an elderly patient.

P. Batty, D. Hart, J. Pasi, 2012, Clinical medicine.

The use of intermediate‐purity clotting factor concentrates and HIV disease progression in men with haemophilia

C. Sabin, P. Lilley, J. Elford, 1996, Haemophilia : the official journal of the World Federation of Hemophilia.

Lack of Activated Protein C Resistance in Healthy Hong Kong Chinese Blood Donors - Correlation with Absence of Arg506-Gln Mutation of Factor V Gene

J. Pasi, H. W. Liu, L. Chan, 1996, Thrombosis and Haemostasis.

Autotransplantation of Unmanipulated Bone Marrow into Scarred Myocardium is Safe and Enhances Cardiac Function in Humans

M. Galiñanes, J. Pasi, D. Chin, 2004, Cell transplantation.

Acute Budd-Chiari syndrome treated by liver transplantation in a woman homozygous for factor V Leiden.

B. Davidson, J. Pasi, K. Rolles, 1996, European Journal of Gastroenterology and Hepathology.

Phenotypic Characterization of EIF2AK4 Mutation Carriers in a Large Cohort of Patients Diagnosed Clinically With Pulmonary Arterial Hypertension

Fengyuan Hu, Kevin Rue-Albrecht, Patrick Deegan, 2017, Circulation.

Diagnostic accuracy study of a factor VIII ELISA for detection of factor VIII antibodies in congenital and acquired haemophilia A

K. Pasi, P. Batty, D. Hart, 2015, Thrombosis and Haemostasis.

Two novel mutations in severe factor VII deficiency

E. Tuddenham, K. Gomez, J. McVey, 2003, British journal of haematology.

Pharmacokinetics, Safety, and Efficacy Of Long-Lasting Recombinant Factor IX Fc Fusion Protein (rFIXFc) In Adolescent Subjects With Hemophilia B: A Subgroup Analysis Of The B-LONG Study

D. Perry, L. Valentino, M. Ozelo, 2013 .

CD4+ Counts before and after Switching to Monoclonal High-Purity Factor VIII Concentrate in HIV-Infected Haemophilic Patients

C. Sabin, Christine A. Lee, G. Janossy, 1994, Thrombosis and Haemostasis.

Comprehensive Rare Variant Analysis via Whole-Genome Sequencing to Determine the Molecular Pathology of Inherited Retinal Disease.

Dorothy A. Thompson, Sri V. V. Deevi, Eleanor F. Dewhurst, 2017, American journal of human genetics.

Activity of Transgene-Produced B-Domain Deleted Factor VIII in Human Plasma Following AAV5 Gene Therapy.

K. Pasi, S. Rangarajan, G. Pierce, 2020, Blood.

Telomerecat: A ploidy-agnostic method for estimating telomere length from whole genome sequencing data

Andy G Lynch, Sri V. V. Deevi, Eleanor F. Dewhurst, 2017, Scientific Reports.

First-in-Human Phase 1/2 Clinical Trial of SIG-001, an Innovative Shielded Cell Therapy Platform, for Hemophilia Α

B. Konkle, A. Shapiro, W. Miesbach, 2020 .

Response to desmopressin is influenced by the genotype and phenotype in type 1 von Willebrand disease (VWD): results from the European Study MCMDM-1VWD.

A. Tosetto, J. Goudemand, S. Lethagen, 2008, Blood.

Prophylaxis with a recombinant factor VIII Fc in hemophilia A: long-term follow-up on joint health, efficacy, and safety from phase 3 studies in children and adults

V. Blanchette, C. Négrier, S. Lethagen, 2023, Research and practice in thrombosis and haemostasis.

Telomerecat: A ploidy-agnostic method for estimating telomere length from whole genome sequencing data

Sri V. V. Deevi, Eleanor F. Dewhurst, Courtney E. French, 2017, Scientific Reports.

A novel variant causing α2 antiplasmin deficiency: case report and experience in a UK centre

S. Sivapalaratnam, D. Hart, J. Pasi, 2019, British journal of haematology.

Biallelic Mutation of ARHGEF18, Involved in the Determination of Epithelial Apicobasal Polarity, Causes Adult-Onset Retinal Degeneration.

Sri V. V. Deevi, Eleanor F. Dewhurst, Lorena E. Lorenzo, 2017, American journal of human genetics.