RAO Network

APBD Research Foundation

Photo of participants in the APBD Tour de Friends UPenn Million Dollar Bike Ride for rare disease research
The APBD Tour de Friends bike team raised funds for research grants by participating in the UPenn Million Dollar Bike Ride for rare disease research. Photo courtesy of APBD Research Foundation.

If you want to go far, go together

Adult polyglucosan body disease (APBD) is a rare inherited disorder that affects the nervous system. Initial symptoms include numbness in the toes and feet and progressive muscle weakness and stiffness. In addition, patients experience urinary frequency that far exceeds the norm. Over time, these symptoms worsen, often leading to a loss of muscle control, and eventually an inability to walk and a near complete loss of urinary control.

Three brothers with this diagnosis and two community advocates saw an opportunity to band together and founded the APBD Research Foundation (APBDRF) in 2005. The Weiss brothers, working with Edwin Kolodny, M.D., and Jeffrey Levenson, DDS, built a scientific advisory board and hosted the first few meetings in Dr. Levenson’s living room. Since then, the foundation has been laser-focused on research, organizing patients and reaching out to allied rare disease groups.

APBDRF has had significant breakthroughs, all made possible by highly collaborative efforts. Having melded science expertise with expressed patient needs, it now has two reliable diagnostic tests, two mouse models of the disease, a neuronal model, and a fibroblast bio-bank. APBDRF is optimistic that it can build on this foundation with a wider pool of collaborators and reach its goals to find a cure and vastly improve the lives of those affected.

APBDRF has had significant breakthroughs, all made possible by highly collaborative efforts.

Learn How We Support Science

Science Funding

Learn more about funding apportunities.

Hear Rare Disease Stories

Hear Rare Disease Stories

Listen to The Story Collider podcast featuring stories about patients who are driving progress against rare disease.