We are excited to share the APBD Research Foundation’s 2023-2025 Strategic Plan. This Strategic Plan is the result of efforts spanning eight months, involving our Board of Directors, members of our Scientific Advisory Board, staff, mentors, and Foundation partners and friends. Please accept our thanks to each of you for your insightful contributions!

In the infographic below, you will see that our Plan sets the course for the work ahead by laying out four overarching strategies.

2023-2025 Strategic Plan

Our Mission

The APBD Research Foundation is the only North American patient advocacy and research organization supporting people with APBD and allied diseases. Our mission is to:

  • Improve the diagnosis and treatment of APBD
  • Support affected individuals and families
  • Increase awareness of APBD among health professionals and the public
  • Facilitate the translation of research into treatments and a cure for APBD

Our Vision

Our vision is “a world without APBD,” accomplished by early diagnosis, accessible care, robust research, reliable information, and effective and affordable treatments.

4 Strategies

Improving Diagnosis and Care

  • Increase clinical knowledge of APBD
  • Support the creation of APBD Centers of Excellence
  • Increase access to information on genetic testing
  • Improve access to patient care

Preparing for Clinical Trials

  • Partner with other polyglucosan storage disease and allied disease organizations
  • Support the establishment and growth of our patient registries
  • Ensure patient voice in every facet of clinical trial development
  • Establish connections with our geographically dispersed patients

Advancing Drug Development

  • Reinvigorate the Scientific and Medical Advisory Board
  • Continue funding research through UPenn’s Million Dollar Bike Ride
  • Expand our network of corporate and academic partners
  • Explore the creation of a research investment fund

Fortifying Internal Operations

  • Hire paid Executive Director
  • Grow the Board of Directors, Scientific and Medical Advisory Board, and staff
  • Implement our Fund Development Plan

We also have important metrics that we are using to track our progress, which we will share in future communications. Please contact us at info@apbdrf.org if you have any questions and would like to learn more about our Plan.

The Plan lays out a bold, but achievable, vision for the future. It builds upon years of work and our recent successes such as the Listening Session with the FDA, the APBD Scientific Conference, the Biomarker Workshops, the APBD and GSD IV Clinical Practice Guidelines, and the study of the global prevalence of APBD and GBE1 disorders. None of them would be possible without you.

With the Plan in place to guide us, the next level of work begins, and it will take community-wide engagement.

Thank you for steadfastly supporting our work to make treatments and a cure a reality. Together, we will ensure a brighter future for patients and families affected by APBD.


Emil Weiss, Co-Founder and Co-President
Jeff Levenson, Co-President
Natacha Pires, Executive Director