selected scholarly activity
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academic article
- Exploring regional variations in the cross‐cultural, international implementation of the Patient Reported Outcomes Burdens and Experience (PROBE) study. Haemophilia. 25:365-372. 2019
- Test-retest properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire and its constituent domains. Haemophilia. 25:75-83. 2019
- The Patient Reported Outcomes, Burdens and Experiences (PROBE) Project: development and evaluation of a questionnaire assessing patient reported outcomes in people with haemophilia. Pilot Feasibility Stud. 4:58. 2018
- Core outcome set for gene therapy in haemophilia: Results of the coreHEM multistakeholder project. Haemophilia. 24:e167-e172. 2018
- Genotypes, phenotypes and whole genome sequence: Approaches from the My Life Our Future haemophilia project. Haemophilia. 24:87-94. 2018
- Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies. Haemophilia. 23:643-644. 2017
- Risk-based decision making and ethical considerations in donor compensation for plasma-derived medicinal products. Transfusion. 56:2889-2894. 2016
- Improving comprehensive care in the haemophilia community: building on the HERO Study. Haemophilia. 22:e320-e322. 2016
- NHF-McMaster Guideline on Care Models for Haemophilia Management. Haemophilia. 22:6-16. 2016
- Assessments of outcome in haemophilia - a patient perspective. Haemophilia. 22:e208-e209. 2016
- The national haemophilia program standards, evaluation and oversight systems in the United States of America.. Blood Transfusion. 12 Suppl 3:e542-e548. 2014
- World Federation of Hemophilia: 50 years of advancing treatment for all. Haemophilia. 19:475-480. 2013
- Factor VIII Products and Inhibitors in Severe Hemophilia A. New England Journal of Medicine. 368:1456-1457. 2013
- Factor VIII products and inhibitors in severe hemophilia A.. New England Journal of Medicine. 368:1456. 2013
- Gene Therapy for Hemophilia: Addressing the Coming Challenges of Affordability and Accessibility. Molecular Therapy. 21:1-2. 2013
- Prospective evaluation of the morbidity and mortality of wild-type and V122I mutant transthyretin amyloid cardiomyopathy: The Transthyretin Amyloidosis Cardiac Study (TRACS). American Heart Journal. 164:222-228.e1. 2012
- Haemophilia care - past, present and future from a patient perspective. Haemophilia. 18:3-5. 2012
- WFH: Closing the global gap - achieving optimal care. Haemophilia. 18:1-12. 2012
- Ensuring maximum outcomes and benefits in comprehensive care for bleeding disorders through surveillance and data collection. Hematology. 17:s147-s149. 2012
- Haemophilia: provision of factors and novel therapies: World Federation of Hemophilia goals and achievements. British Journal of Haematology. 154:704-714. 2011
- Building our global family - achieving treatment for all. Haemophilia. 16:1-10. 2010
- Global data and haemophilia care trends: commentary. Haemophilia. 16:18-19. 2010
- WFH – the cornerstone of global development: 45 years of progress. Haemophilia. 14:1-9. 2008
- Treatment for all: a vision for the future. Haemophilia. 12:169-173. 2006
- More on: are randomized clinical trials the only truth? Not always. Journal of Thrombosis and Haemostasis. 4:1167-1168. 2006
- What is a cure and how do we get there?. Haemophilia. 10:115-118. 2004
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conference paper
- "Problem Joint" a more patient relevant definition for joint morbidity in haemophilia. Haemophilia. 111-112. 2019
- Impact of acute and chronic pain on the EQ-5D: Insights from the PROBE study. Haemophilia. 75-76. 2019
- Part-time employment and early retirement in people with severe haemophilia: Insights from the PROBE study. Haemophilia. 137-138. 2019
- Physical activity in persons with hemophilia (PwH) from France (FR), Italy (ITA), and United States (US) from the hemactive patient survey. Haemophilia. 78-78. 2019
- Predictors for acute and chronic pain in patients with severe haemophilia in the PROBE cohort. Haemophilia. 76-76. 2019
- First Steps in Choosing Appropriate Instruments/Measurements for a Core Outcome Set: An Example from Corehem. Value in Health. S212-S212. 2018
- The PROBE Data Dashboard - A new tool for real-time data visualization and analysis of patient reported outcomes. Haemophilia. 181-182. 2018
- The PROBE Study - Global burden of bleeding from the mouth in Haemophilia - a call to action. Haemophilia. 66-67. 2018
- coreHEM: A multi-stakeholder approach to developing a core outcome set for gene therapy in hemophilia. Haemophilia. 81-81. 2018
- Occurrence and impact of pain among patients living with hemophilia: An analysis from the patient reported outcomes, burdens and experiences (PROBE) study. Haemophilia. 26-27. 2018
- Estimating The Global Annual Bleed Rate In Haemophilia. Haemophilia. 86-86. 2017
- Test-Retest Reliability Analysis of The Patient Reported Outcomes Burdens and Experiences (PROBE) Study. Haemophilia. 50-51. 2017
- Test-Retest Reliability Analysis of the Patient Reported Outcomes Burdens and Experiences (PROBE). Blood. 2016
- NHF-MCMASTER GUIDELINE ON CARE MODELS FOR HEMOPHILIA MANAGEMENT. American Journal of Hematology. E428-E429. 2016
- A risk based decision making framework - stakeholder consultation essential to appropriate minimization of blood safety risk. Haemophilia. 132-132. 2016
- Development of a questionnaire to identify hemophilia A patient and clinician discussion opportunities around prophylaxis treatment options. Haemophilia. 31-31. 2016
- The patient reported outcomes burdens and experiences (PROBE) study phase 1 methodology and feasibility results. Haemophilia. 118-119. 2016
- The patient reported outcome burdens and experiences (PROBE) study - phase 1 results show PROBE study methodology feasible. Journal of Thrombosis and Haemostasis. 47-47. 2016
- The Patient Reported Outcomes, Burdens, and Experiences (Probe) Phase 1 Study Methodology and Feasibility. Haemophilia. 43-44. 2016
- Cultivating and Preparing Future Leaders. Haemophilia. 185-185. 2014
- Speaking as one voice: Uniting and educating advocates. Haemophilia. 123-124. 2014
- The Rise of the Patient Voice. Haemophilia. 179-179. 2014
- Hemophilia and von Willebrand disease patients' perceptions of care at US Hemophilia Treatment Centers. Haemophilia. 13-13. 2014
- Morbidity and Mortality of Transthyretin (TTR) Amyloid Cardiomyopathy (ATTR-CM): Transthyretin Amyloidosis Cardiac Study (TRACS) a Prospective Evaluation. Circulation. S717-S717. 2008