The MRCT Center Podcast

EP 5 - Navigating the MRCT Center’s Long-Term Follow-Up Toolkit for Gene Therapies

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Episode Overview:

In this fifth episode of the MRCT Center podcast, Dr. Barbara Bierer, Faculty Director of the MRCT Center and professor of medicine at BWH and HMS, discusses the newly released Toolkit for Supporting the Design, Conduct, and Reporting of Long-Term Follow-Up (LTFU) Studies for Gene Therapies with Carolyn Chapman, Leader of the Cell and Gene therapies project at the MRCT Center, Lead Investigator at BWH and member of the faculty at HMS.


Key Discussion Topics:

  • What are Long-Term Follow-up studies for gene therapies and why are they needed
  • Who the LTFU toolkit designed for
  • The development of the toolkit through diverse collaboration
  • The timeliness and importance of this toolkit release
  • A comprehensive walk-through of the sections and features of the toolkit
  • What the porridge is the "Goldilocks issue"?
  • In depth discussion on what it means for a patient to be part of a LTFU study and how to address the potential burdens


    • Learn More:
    Access the full LTFU Toolkit and additional standalone resources at:
    mrctcenter.org/LTFUToolkit


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    The MRCT Center PodcastBy The MRCT Center