We are excited to announce that the GFPD has been awarded a grant to undertake a Natural History Study project with the National Organization for Rare Disorders (NORD), funded in cooperation with the Food and Drug Administration (FDA). Our Primary Investigator, GFPD Medical and Research Liaison Dr. Mousumi Bose, will work with a Scientific Advisory team including Dr. Nancy Braverman of McGill University, Dr. Joe Hacia of University of Southern California, and Jessie Kulaga-Yoskovitz of McGill University. Together with this team, the GFPD will build a database of experiences and outcomes for patients with Peroxisome Biogenesis Disorder – Zellweger Spectrum Disorder (PBD-ZSD).
The importance of the natural history study as a research tool cannot be overstated. Dr. Bose explains that “when patients with rare diseases are given the opportunity to connect and compare stories about symptoms, experiences with particular therapies, and their individual course with PBD-ZSD, a clearer picture of the disease begins to emerge, which scientists can use in shaping meaningful research.”
NORD selected twenty patient organizations in a “competitive application process,” and notes that the “diseases represented have diagnostic challenges, limited or no research, and cover a broad range of symptoms and medical specialties, including neurology, cardiovascular, musculoskeletal, immunology and endocrinology.” Undertaking this important project in collaboration with NORD and with a cohort of other rare disease patient organizations will benefit not only PBD-ZSD research but other rare disease populations as well.
We’re looking forward to getting to work on this project and will seek to begin engaging parents of patients affected by PBD-ZSD in the weeks and months ahead.
For more, read NORD’s press release.