selected scholarly activity
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books
- Geographical Gerontology 2017
- Preface. Ed. 56. 2015
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chapters
- Geographical gerontology: Progress and possibilities. 313-319. 2017
- Space and place in geographical gerontology: Theoretical traditions, formations of hope. 11-28. 2017
- Ageing Resource Communities. 206-212. 2015
- Perspectives on rural voluntarism. 146-148. 2015
- Aging and Older People, Geography of. 407-412. 2015
- Comprehensive care and delivery of care: the global perspective. 515-521. 2014
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conferences
- Test–retest reliability of a mobile application of the patient reported outcomes burdens and experiences (PROBE) study. Haemophilia. 702-708. 2024
- Initial results of the impact of valoctocogene roxaparvovec on pain occurrence and interference: Insights from PROBE. Haemophilia. 20-22. 2023
- A COMPARATIVE STUDY OF DIFFERENT METHODS OF HANDLING MISSING DATA IN PATIENT REPORTED OUTCOMES, BURDENS AND EXPERIENCES (PROBE) SCORE ALGORITHM AMONG PEOPLE WITH HEMOPHILIA. American Journal of Hematology. E48-E48. 2023
- COLLABORATION FOR THE LONG-TERM FOLLOW-UP OF PEOPLE WITH HEMOPHILIA TREATED WITH GENE THERAPY: WORLD FEDERATION OF HEMOPHILIA- AMERICAN THROMBOSIS AND HEMOSTASIS NETWORK. American Journal of Hematology. E23-E23. 2023
- NATIONAL ECONOMIC BURDEN OF ADULTS WITH SEVERE HEMOPHILIA A MANAGED WITH PROPHYLAXIS IN THE UNITED STATES. American Journal of Hematology. E118-E118. 2023
- A Novel Methodology for Building Longitudinal, PatientCentric Real World Datasets in Hemophilia A. Blood. 2021
- Relationship of EQ-5D Pain Domain and Bleeds - Insights from the PROBE Study. Haemophilia. 12-12. 2021
- THE WORLD FEDERATION OF HEMOPHILIA GENE THERAPY REGISTRY. Haemophilia. 132-133. 2021
- Examination and Validation of a Patient-Centric Joint Metric: "Problem Joint"; Empirical Evidence from the CHESS US Dataset. Blood. 25-26. 2020
- Developing a patient reported outcome measure to explore mental health outlook in people with Hemophilia: Development of a conceptual framework. Haemophilia. 62-62. 2020
- Education in people with Hemophilia - Insights from the PROBE Study. Haemophilia. 132-132. 2020
- Impact of Pain on Physical Activity by Persons with Hemophilia (PwH) from the HemACTIVE Patient Survey. Haemophilia. 118-118. 2020
- International Standard Set of Health Outcomes that matter for people living with hemophilia. Haemophilia. 60-61. 2020
- Quantifying the disability paradox in hemophilia. Haemophilia. 130-131. 2020
- Treatment adherence in people with hemophilia (PwH) who are restricted by pain: Findings from the HemACTIVE Patient Survey. Haemophilia. 81-81. 2020
- DEVELOPMENT AND TESTING OF THE ONLINE PROBE SURVEY AND THE MYPROBE APP AND INTEGRATION WITH THE CANADIAN BLEEDING DISORDER REGISTRY. Haemophilia. 76-77. 2020
- coreHEM, a Multistakeholder Core Outcome Set Project for Gene Therapy in Hemophilia. Molecular Therapy. 277-277. 2019
- "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
- Burden of Comorbid Diseases in Patients with Hemophilia: The Cross-Sectional Analysis of the Patient Reported Outcomes, Burden and Experiences (PROBE) Study. Blood. 2017
- Sexual Health in Patients with Hemophilia; The Insights from the Patient Reported Outcomes, Burdens and Experiences (PROBE) Study. Blood. 2017
- 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). Blood. 5964-5964. 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
- Global Forum of the World Federation of Hemophilia, September 26–27, 2005, Montreal, Quebec, Canada. Transfusion and Apheresis Science. 151-172. 2006
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journal articles
- Recommending defunct therapies is a safety risk for hemophilia.. Blood Transfusion. 23:189-190. 2025
- Pain-Related Quality of Life Outcomes in People With Haemophilia A Receiving Emicizumab: A Post Hoc Analysis of the HAVEN 1, 3 and 4 and STASEY Studies.. Haemophilia. 31:87-98. 2025
- Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and Von Willebrand Disease.. Haemophilia. 2024
- International Society on Thrombosis and Haemostasis Clinical Practice Guideline for Treatment of Congenital Haemophilia-A Critical Appraisal.. Haemophilia. 2024
- A core outcome set for prophylaxis and perioperative treatment of von Willebrand disease: The coreVWD initiative.. Haemophilia. 30:1357-1365. 2024
- Development of the World Federation of Hemophilia Shared Decision-Making Tool.. Haemophilia. 30:1298-1308. 2024
- Development of the coreHEM mental health patient-reported outcome measure - A novel mental health outlook measure for people with haemophilia.. Haemophilia. 30:1309-1320. 2024
- Risk of harm to people with haemophilia from the 2023 WHO Essential Medicines List. The Lancet Haematology. 11:e638-e640. 2024
- Association of patient, treatment and disease characteristics with patient‐reported outcomes: Results of the ECHO Registry. Haemophilia. 30:106-115. 2024
- Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1. Journal of Thrombosis and Haemostasis. 21:3450-3462. 2023
- Hemophilia gene therapy: first, do no harm. Journal of Thrombosis and Haemostasis. 21:2354-2361. 2023
- Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities in health services; diversity, equity, and inclusion; and implementation science. Expert Review of Hematology. 16:87-106. 2023
- Data sharing to advance gene‐targeted therapies in rare diseases. American Journal of Medical Genetics - Seminars in Medical Genetics. 193:87-98. 2023
- Gene‐targeted therapies: Towards equitable development, diagnosis, and access. American Journal of Medical Genetics - Seminars in Medical Genetics. 193:56-63. 2023
- Humanistic burden of problem joints for children and adults with haemophilia. Haemophilia. 29:608-618. 2023
- Prophylaxis use of clotting factor replacement products in people with non‐severe haemophilia: A review of the literature. Haemophilia. 29:33-44. 2023
- Association of factor expression levels with health-related quality of life and direct medical costs for people with haemophilia B. Journal of Medical Economics. 25:386-392. 2022
- Achieving access to haemophilia care in low-income and lower-middle-income countries: expanded Humanitarian Aid Program of the World Federation of Hemophilia after 5 years. The Lancet Haematology. 9:e689-e697. 2022
- User-Centered Development and Testing of the Online Patient-Reported Outcomes, Burdens, and Experiences (PROBE) Survey and the myPROBE App and Integration With the Canadian Bleeding Disorder Registry: Mixed Methods Study. JMIR Human Factors. 9:e30797-e30797. 2022
- A preliminary application of a haemophilia value framework to emerging therapies in haemophilia. Haemophilia. 28:9-18. 2022
- The Hemophilia Gene Therapy Patient Journey: Questions and Answers for Shared Decision-Making. Patient Preference and Adherence. Volume 16:1439-1447. 2022
- Eliminating Panglossian thinking in development of AAV therapeutics. Molecular Therapy. 29:3325-3327. 2021
- Evaluation of the sexual health in people living with hemophilia. Haemophilia. 27:993-1001. 2021
- Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment. Haemophilia. 27:769-782. 2021
- The effect of emicizumab prophylaxis on long‐term, self‐reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies. Haemophilia. 27:854-865. 2021
- Evolution of haemophilia integrated care in the era of gene therapy: Treatment centre’s readiness in United States and EU. Haemophilia. 27:511-514. 2021
- Vaccination against COVID‐19: Rationale, modalities and precautions for patients with haemophilia and other inherited bleeding disorders. Haemophilia. 27:515-518. 2021
- Patient‐relevant health outcomes for hemophilia care: Development of an international standard outcomes set. Research and Practice in Thrombosis and Haemostasis. 5:e12488-e12488. 2021
- Evidence of a disability paradox in patient‐reported outcomes in haemophilia. Haemophilia. 27:245-252. 2021
- Identified unmet needs and proposed solutions in mild‐to‐moderate haemophilia: A summary of opinions from a roundtable of haemophilia experts. Haemophilia. 27:25-32. 2021
- Integrated Hemophilia Patient Care via a National Network of Care Centers in the United States: A Model for Rare Coagulation Disorders. Journal of Blood Medicine. Volume 12:897-911. 2021
- Non‐severe haemophilia: Is it benign? – Insights from the PROBE study. Haemophilia. 27:17-24. 2021
- Personalising haemophilia management with shared decision making. The Journal of Haemophilia Practice. 8:69-79. 2021
- The potential impact of gene therapy on health-related quality of life (HRQoL) domains in haemophilia. The Journal of Haemophilia Practice. 8:56-68. 2021
- Challenges and key lessons from the design and implementation of an international haemophilia registry supported by a pharmaceutical company. Haemophilia. 26:966-974. 2020
- Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH. Journal of Thrombosis and Haemostasis. 18:3074-3077. 2020
- Gene therapy to cure haemophilia: Is robust scientific inquiry the missing factor?. Haemophilia. 26:931-933. 2020
- The role of telemedicine in the delivery of health care in the COVID‐19 pandemic. Haemophilia. 26:e230-e231. 2020
- The World Federation of Hemophilia Annual Global Survey 1999‐2018. Haemophilia. 26:591-600. 2020
- World Federation of Hemophilia Gene Therapy Registry. Haemophilia. 26:563-564. 2020
- Achieving the unimaginable: Health equity in haemophilia. Haemophilia. 26:17-24. 2020
- An international survey to inform priorities for new guidelines on von Willebrand disease. Haemophilia. 26:106-116. 2020
- Exploring regional variations in the cross‐cultural, international implementation of the Patient Reported Outcomes Burdens and Experience (PROBE) study. Haemophilia. 25:365-372. 2019
- Hemophilia trials in the twenty‐first century: Defining patient important outcomes. Research and Practice in Thrombosis and Haemostasis. 3:184-192. 2019
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The 1st
WFH Gene Therapy Round Table: Understanding the landscape and challenges of gene therapy for haemophilia around the world. Haemophilia. 25:189-194. 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 and Feasibility Studies. 4:58. 2018
- Aging Through the Lens of Historical Time, Space, and Place. The Gerontologist. 58:795-796. 2018
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Core outcome set for gene therapy in haemophilia: Results of the core
HEM 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
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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
- Cell Phones and Landlines: The Impact of Gene Therapy on the Cost and Availability of Treatment for Hemophilia. Molecular Therapy. 19:1749-1750. 2011
- 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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preprints