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International Intestinal Failure Registry and The FDA Grant 101

0.0 - Information Session for Caregivers and Patients

Presenter: Yaron Avitzur, Simon Horslen, , ,
Authors: Yaron Avitzur, Simon Horslen, Christina Belza, Marie Neumann

Overview

Intestinal failure is a rare disease with challenging management strategies. Research is critically important to advancing the field and informing practice and engaging with patients and caregivers to facilitate current and future research initiatives is imperative. The session will provide a clear, accessible overview of the registry’s purpose and its role in advancing knowledge of long-term outcomes, treatment effectiveness, and patient-centred priorities in intestinal failure. It will also outline how registry data may inform clinical care, support research innovation, and contribute to improved therapies and health system planning. A goal of the webinar will be the importance of family engagement in research, emphasizing patients and caregivers as active partners whose lived experiences help shape research priorities, outcome selection, and study design. The session will promote opportunities for meaningful involvement through registry participation and advisory roles, while fostering transparency, trust, and open dialogue through an interactive question-and-answer component.

Learning Objectives

  • Will provide a clear, accessible overview of the registry’s purpose, including its role in advancing understanding of long-term outcomes, treatment effectiveness, and patient-centered priorities in intestinal failure
  • Will outline how registry data may inform clinical care, support research innovation, and contribute to improved therapies and health system planning
  • Will highlight the goals and scope of the FDA grant, including its focus on strengthening data infrastructure and accelerating meaningful research in rare pediatric conditions

Participants

Christina Belza, Canada
Moderator
Bio
Marie Neumann, United States
Moderator
Bio
Yaron Avitzur, Canada
Speaker
Bio
Simon Horslen, United States 
Speaker
Bio

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  5. The information cannot be used as a substitute for professional care.
  6. The information does not represent a standard of care.
  7. No physician-patient relationship is being established.

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