Foundation Approves and Implements its 2023-2025 Strategic Plan

Dear Friend,

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.

Sincerely,

Emil Weiss, Co-Founder and Co-President

Jeff Levenson, Co-President

Natacha Pires, Executive Director

Art Glassman and Beth Katz Join the APBD Research Foundation’s Board of Directors

The APBD Research Foundation is delighted to announce that Art Glassman and Beth Katz have joined the Board of Directors. “As we work to implement the Foundation’s new strategic plan, Art and Beth will provide valuable input and guidance as new members of the Board,”

shared Emil Weiss, co-founder, and co-president.

Click here to read more...

Raising APBD Research Funds at the Million Dollar Bike Ride!

To date, we have raised over $32,800 in anticipation of the Million Dollar Bike Ride for rare disease research fundraising program. And, we still have a few days to go!

The first $30,000 of your gifts will be matched by UPenn's Orphan Disease Center and again by generous APBD community supporters for a total of $92,800 in APBD research grant funding for 2024. Can we make the grand total hit $100,000?

Haven’t made your gift to support APBD research yet? Click here to give…

You can also participate in the Million Dollar Mile!

The event now has the option to participate in the Million Dollar Mile walk in Philadelphia on June 10. This option is perfect for kids, strollers, and folks with mobility aids.

Click here to register for free…

Upcoming Events

From Chat events to workshops and scientific symposiums, the Foundation hosts programs for patients, caregivers, and health professionals which connect our community members and deepen the understanding of how APBD impacts lives.

These events offer peer support, opportunities to hear from health experts and Foundation leaders, and the chance to raise funds for APBD research.