ATHN Researchers

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B

Author(s):

Steven W Pipe, Frank W G Leebeek, Michael Recht, Nigel S Key, Giancarlo Castaman, Wolfgang Miesbach, Susan Lattimore, Kathelijne Peerlinck, Paul Van der Valk, Michiel Coppens, Peter Kampmann, Karina Meijer, Niamh O’Connell, K John Pasi, Daniel P Hart, Rashid Kazmi, Jan Astermark, Cedric R J R Hermans, Robert Klamroth, Richard Lemons, Nathan Visweshwar, Annette von Drygalski, Guy Young, Shelley E Crary, Miguel Escobar, Esteban Gomez, Rebecca Kruse-Jarres, Doris V Quon, Emily Symington, Michael Wang, Allison P Wheeler, Robert Gut, Ying P Liu, Ricardo E Dolmetsch, David L Cooper, Yanyan Li, Brahm Goldstein, Paul E Monahan

Publication:

New England Journal of Medicine

Date:

February 23, 2023

Topics:

Hemophilia B, Gene Therapy, Etranacogene Dezaparvovec

Building the Blueprint: Formulating a Community-Generated National Plan for Future Research in Inherited Bleeding Disorders

Author(s):

Leonard Valentino, Michelle Witkop, Maria Santaella, Donna DiMichele, Michael Recht

Publication:

Haemophilia

Date:

June 14, 2022

Topics:

Community, Blueprint Bleeding Disorders

Eptacog Beta Efficacy and Safety in the Treatment and Control of Bleeding in Paediatric Subjects (<12 Years) with Haemophilia A or B with Inhibitors

Author(s):

Steven Pipe, Cedric Hermans, Meera Chitlur, Manuel Carcao, Giancarlo Castaman, Joanna Davis, Jonathan Ducore, Amy Dunn, Miguel Escobar, Janna Journeycake, Osman Khan, Johnny Mahlangu, Shannon Meeks, Ismail Haroon Mitha, Claude Negrier, Ulrike Nowak-Gottl, Michael Recht, Tammuella Christenery-Singleton, Olekasandra Stasysyn, Katerna Vilchevska, Laura Villarreal Martinez, Michael Wang, Jerzy Windyga, Guy Young, W Allan Alexander, Daniel Bonzo, Christopher Macie, Ian Mitchell, Evelyne Sauty, Thomas Al Wilkinson, Amy D Shapiro

Publication:

Haemophilia

Date:

April 27, 2022

Topics:

Eptacog, Paediatric, Safety and Efficacy, Hemophilia A or B Inhibitors

Incorporating the Patient Voice and Patient Engagement in GOAL-Hem: Advancing Patient-Centric Hemophilia Care

Author(s):

Jonathan C Roberts, Michael Recht, Sarah E Gonzales, Justin Stanley, Michael Denne, Jorge Caicedo, Kenneth Rockwood

Publication:

Research and Practice in Thrombosis and Haemostasis

Date:

February 7, 2022

Topics:

goals, hemophilia

Iron Ladies: Variation in the Identification and Management of Iron Deficiency in Women with Bleeding Disorders

Author(s):

Meghan McCormick, Margaret Ragni

Publication:

Blood

Date:

November 23, 2021

Topics:

Identification, ID (Iron Deficiency), Women

Hemophilia Gene Therapy Value Assessment: Methodological Challenges and Recommendations

Author(s):

Louis P Garrison, Ed Pezalla, Adrian Towse, Hongbo Yang, Elizabeth Faust, Eric Q Wu, Naxin Li, Eileen Sawyer, Michael Recht

Publication:

Value in Health

Date:

November 1, 2021

Topics:

Hemophilia, Gene Therapy, Assessment

A Prospective Observational Study of Antihemophilic Factor (recombinant) Prophylaxis Related to Physical Activity Levels in Patients with Hemophilia A in the United States (SPACE)

Author(s):

Barbara A Konkle, Doris V Quon, Leslie Raffini, Michael Recht, Vlad C Radulescu, Shannon L Carpenter, Amy L Dunn, Mei Lu, Maureen Watt

Publication:

Journal of Blood Medicine

Date:

October 14, 2021

Topics:

Prophylaxis, Physical Activity Levels

A Delphi Consensus Approach for Difficult-to-Treat Patients with Severe Hemophilia A without Inhibitors

Author(s):

Sreenivas P Veeranki, Priti Pednekar, Marlon Graf, Rifat Tuly, Michael Recht, Katharine Batt

Publication:

Journal of Blood Medicine

Date:

October 1, 2021

Topics:

Treatments, Severe Hemophilia

Understanding racial differences in attitudes about public health efforts during COVID-19 using an explanatory mixed methods design

Author(s):

Paige Nong, Minakshi Raj, Marie Grace Trinidad, Zachary Rowe, Jodyn Platt

Publication:

Social Science & Medicine

Date:

October 2021

Topics:

Health Equity

NHF Builds a Community-Driven National Research Blueprint for Inherited Bleeding Disorders

Author(s):

Michelle Witkop, Michael Recht, Donna DiMichele, Leonard Valentino

Publication:

National Hemophilia Foundation

Date:

September 2021

Topics:

NHF, Research Blueprint, Blood Disorder

Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment

Author(s):

Michelle Witkop, George Morgan, Jamie O’Hara, Michael Recht, Tyler Buckner, Diane Nugent, Randall Curtis, Brian O’Mahony, Mark Skinner, Brendan Mulhern, Matthew Cawson, Talaha Ali, Eileen Sawyer, Naxin Li

Publication:

Haemophilia

Date:

July 26, 2021

Topics:

Hemophilia, Gene Therapy

Bounded Justice and the Limits of Health Equity

Author(s):

Melissa Creary

Publication:

Cambridge University Press

Date:

June 29, 2021

Topics:

Health Equity

Adult lifetime cost of hemophilia B management in the US: Payer and societal perspectives from a decision analytic model

Author(s):

Naxin Li, Eileen Sawyer, Konrad Maruszczyk, Greg Guzauskas, Marta T Slomka, Tom Burke, Anthony P Martin, Jaime O’Hara, Matt Stevenson, Michael Recht

Publication:

Journal of Medical Economics

Date:

Jan-Dec 2021

Topics:

Hemophilia

A Study of Dedicated Haemophilia Carrier Clinics in the United States: Prevalence, Services Offered, and Barriers to Development

Author(s):

Ariela L Marshall, Michael Recht, Meera Sridharan, William L Nichols, Aneel A Ashrani, Karen M Fischer, Moses Miles, Angela Riedel, Rajiv K Pruthi

Publication:

WHF Virtual Summit

Date:

June 14-19, 2020

Topics:

Hemophilia, Prevalence, Barriers

The Critical Need for Post-Marketing Surveillance in Gene Therapy for Haemophilia

Author(s):

Barbara Konkle, Michael Recht, Anneliese Higer, Peter Marks

Publication:

Haemophilia Supplemental Article

Date:

June 3, 2020

Topics:

Hemophilia, Gene Therapy

ATHN CV

ATHN Researchers

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B

Building the Blueprint: Formulating a Community-Generated National Plan for Future Research in Inherited Bleeding Disorders

Eptacog Beta Efficacy and Safety in the Treatment and Control of Bleeding in Paediatric Subjects (<12 Years) with Haemophilia A or B with Inhibitors

Incorporating the Patient Voice and Patient Engagement in GOAL-Hem: Advancing Patient-Centric Hemophilia Care

Iron Ladies: Variation in the Identification and Management of Iron Deficiency in Women with Bleeding Disorders

Hemophilia Gene Therapy Value Assessment: Methodological Challenges and Recommendations

A Prospective Observational Study of Antihemophilic Factor (recombinant) Prophylaxis Related to Physical Activity Levels in Patients with Hemophilia A in the United States (SPACE)

A Delphi Consensus Approach for Difficult-to-Treat Patients with Severe Hemophilia A without Inhibitors

Understanding racial differences in attitudes about public health efforts during COVID-19 using an explanatory mixed methods design

NHF Builds a Community-Driven National Research Blueprint for Inherited Bleeding Disorders

Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment

Bounded Justice and the Limits of Health Equity

Adult lifetime cost of hemophilia B management in the US: Payer and societal perspectives from a decision analytic model

A Study of Dedicated Haemophilia Carrier Clinics in the United States: Prevalence, Services Offered, and Barriers to Development

The Critical Need for Post-Marketing Surveillance in Gene Therapy for Haemophilia