This episode of the Global Hemophilia Report podcast explores the significance of grassroots advocacy within hemophilia associations and chapters, emphasizing the critical role of data in advancing advocacy efforts. Experts from various U.S. organizations discuss the historical impact of the 1980s blood contamination crisis and the evolution of advocacy through its challenges and triumphs. The conversation highlights the importance of data collection, transparency, and community engagement in sustaining and enhancing treatment and support for those affected by hemophilia. The episode also addresses future advocacy strategies and the importance of continuous collaboration at the national and local levels.

Contributors:

Rigo Garcia, Hemophilia Federation of Southern California

Sue Lerch, Great Lakes VE HTC

Sue Martin, Bleeding Disorders Association of South Carolina

Linda Mugford, Hemophilia Association of New York, Inc.

Ray Stanhope, Lone Star Bleeding Disorders Foundation

Senior Advisor: Donna DiMichele, MD

Hosted & Written by: Patrick James Lynch

Featured Advertiser:

Sanofi

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Show Notes:

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On this episode we talk with experts Dr. Amy Shapiro, Dr. Maria Elisa Mancuso, Dr. Steve Pipe, Dr. Johnny Mahlangu, and Dr. Lynn Malec to delve into the ongoing evolution of hemophilia therapies. The discussion highlights recent advancements in treatments such as emicizumab, extended half-life factor VIII therapies, and investigates medications in clinical trials like Concizumab, Marstacimab, and Fitusiran. The episode also reflects on the role of treatment individualization and the need for more inclusive research data.

Contributors:

Johnny Mahlangu, MBBCh, MMed, FCPath

Lynn Malec, MD, MSc

Elisa Mancuso, MD

Steven Pipe, MD

Amy Shapiro, MD

Senior Advisor: Donna DiMichele, MD

Hosted & Written by: Patrick James Lynch

Featured Advertiser:

Sanofi

Subscribe to the Global Hemophilia Report

Show Notes:

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On this episode of the Global Hemophilia Report, Senior Advisor Dr. Donna DiMichele leads a comprehensive discussion on FVIII inhibitors—a critical complication in Hemophilia A. The episode features experts such as Dr. Kathleen Pratt, Dr. David Lillicrap, Dr. Bhavya Doshi, Dr. Carol Miao, and Dr. Radek Kazmarek who explore the intricacies of immune responses, gene therapy, and novel therapies like emicizumab. They delve into factors like antigen-presenting cells, B cells, T cells, microbiome effects, and glycans that influence inhibitor development and tolerance. Despite advancements in treatment, the experts underscore the enduring importance of ongoing research in understanding and preventing FVIII inhibitors.

Contributors:

Glaivy Batsuli, MD

Bhavya Doshi, MD

David Lillicrap, MD

Carol Miao, PhD

Kathleen Pratt, PhD

Radek Kaczmarek, PhD

Senior Advisor: Donna DiMichele, MD

Hosted & Written by: Patrick James Lynch

Featured Advertiser:

Sanofi

Subscribe to the Global Hemophilia Report

Show Notes:

Listen to the Global Hemophilia Report Episode 1: Inhibitors: Prevention, Eradication, and Lived Experiences

Conflicts of Interest: Dr. Kathleen Pratt is an inventor on patents related to factor VIII immunogenicity, and she has received research funding in the past from pharmaceutical companies that manufacture human FVIII. She has no other relevant conflicts to report.

Disclaimer: The contents of Dr. Pratt's presentation are the sole responsibility of the speaker and do not necessarily reflect the views, opinions or policies of Uniformed Services University of the Health Sciences (USUHS), The Henry M. Jackson Foundation for the Advancement of Military Medicine, Inc., the Department of Defense (DoD) or the Departments of the Army, Navy, or Air Force. Mention of trade names, commercial products, or organizations does not imply endorsement by the U.S. Government.

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