The work of the GFPD is focused on family, research, and hope. Family-centered activities like support groups, an equipment exchange, a biennial family conference, annual regional meetups, as well as access to researchers and physicians help our families cope with the daily challenges of caring for a child with a terminal disease. However, to cope alone, is not enough. We promote hope for families by actively pursuing the development of therapeutic treatments with our partners at the National Institutes of Health (NIH), USC Keck School of Medicine and McGill University. Additionally, we fund research grants, coordinate our annual scientific conference, collaborate on projects with other organizations in the rare disease space, host a family registry, and publish treatment guidelines.

2017 Highlights

Community Advisory Council and Scientific Initiatives Committee established in February
2017.
 Family and Scientific Conference, featuring Francis Collins, Director of National Institutes
of Health as Keynote Speaker, held in Washington, D.C. in July 13th-17th 2017. Over 110
scientists and physicians along with nearly 200 family members gathered together to
collaborate and seek treatments for peroxisomal disorders.
 Development of a new mouse model with PBD-ZSD at Jackson Laboratories to help test
treatments for the central nervous system component of PBD-ZSD.
 Collaborations with the National Organization for Rare Diseases (NORD) and Global
Genes to extend our reach in the rare disease space.
 Supported research by funding a fellowship at the National Center for Advancing
Translational Sciences. This collaboration with NCATS will fund a post-doctoral position
to run high throughput drug screening, which will search through catalogs of thousands
of already FDA approved drugs and compounds to see if any improve peroxisome
function.

We have a strong staff and committee structure. Our current committees include: Governance, Finance, Community Advisory Council, Scientific Initiatives Committee, Scientific Advisory Board, and Development. Our board members and volunteers are actively involved in the ensuring the mission of the GFPD is accomplished. We have supportive families in our community who are committed to annual fundraisers to benefit the GFPD to help further our mission as well.

We have program evaluation tools for both family and scientific initiatives.

The GFPD was founded on October 5th, 2010 by a small but dedicated group of parents of
children affected by peroxisomal biogenesis disorder.  These parents (our first Board members)
were passionate about finding ways to improve the lives of patients with peroxisomal disorders
and promote and fund research to develop a greater understanding of peroxisomal disorders.

Since 2010, we have grown our patient registry from 48 patients, to today serving over 400
families worldwide who have a living or deceased child with a peroxisomal disorder. Today, our
Board consists of Tulsa, Oklahoma, based professionals as well as parents of children with a
peroxisomal disorder.

Future plans include expanding our family support initiatives and continuing to work towards robust treatment options for peroxisomal disorders.