Season’s Greetings from the Global Foundation for Peroxisomal Disorders!
We are wrapping up a busy and productive year in which we have hosted events, supported families, and promoted scientific research in the field of Peroxisomal Biogenesis Disorder-Zellweger Spectrum Disorder (PBD-ZSD). We traveled to the National Institutes of Health in Bethesda, MD, to tour the labs of the National Center for Translational Sciences, where the GFPD is funding a multi-year research project in search of potential treatments for patients with PBD-ZSD. Additionally, we hosted our annual scientific planning meeting, where we welcomed members of our Scientific Advisory Board as well as members of the research community at large, to discuss potential treatments for PBD-ZSD.
Like you, we are committed to promoting research that will result in therapeutic treatments for our children. We are excited about the GFPD’s role in the development of a new mouse model for PBD-ZSD. This new mouse model will significantly add to the understanding of PBD-ZSD as it will exhibit central nervous system (CNS) dysfunction similar to the neurological symptoms seen in patients with PBD-ZSD. An earlier PBD-ZSD mouse model (funded by the Hopkins family in 2009) has been used to study liver and eye disease, but not CNS symptoms.
This new mouse model of PBD-ZSD is a vital first step in generating innovative treatment options for PBD-ZSD patients experiencing devastating neurological symptoms such as a progressive leukodystrophy or uncontrolled seizures. To make this and other crucial research projects a reality, we need your financial support!
Even with these exciting research opportunities in the works, family support remains a vital part of the GFPD’s success. The GFPD supports families on a daily basis through online parent support groups, an equipment exchange, and assistance with medical and educational advocacy. The involvement of our families is essential to fostering developments in the scientific field. We are supporting the collection of natural history data to document the medical and quality of life experiences of patients with PBD-ZSD. Through the continued collaboration of our families and our devoted scientific community, we hope someday to have better treatment options for children diagnosed with this devastating disease.
All that we have been able to achieve this year has been made possible by financial contributions from our community. Thank you for partnering with us as we fulfill our mission of supporting families and furthering research on PBD-ZSD.
Melissa Bryce Gamble
Mother of Ginny PBD-ZSD (8.5.2008-4.25.2015)
President and Co-Founder, GFPD
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