MISSION AND OVERVIEW
Partners For Krabbe Research (P4KR) is a 501(c)3 foundation. The mission is to increase awareness and to support research to improve the lives of those born with Krabbe disease.
Partners For Krabbe Research is dedicated to engaging with researchers, clinicians, families, other foundations associated with Krabbe and other Leukodystrophies, to support the need for an effective treatment for patients affected by Krabbe disease. By supporting research through fundraising opportunities, P4KR will donate funds to researchers, with the hope of wiping out the devastating symptoms of Krabbe disease.
Through all forms of social media, internet communications and other networking relationships, P4KR will create and develop opportunities to educate and create awareness about Krabbe disease. By establishing a network to service the Krabbe community, P4KR will utilize every resource available not only to find support for patients, but to also ease the pain and suffering that Krabbe disease brings to children and families affected with this disease.
To ultimately find a cure for Krabbe disease.
Partners For Krabbe Research (P4KR) is a volunteer foundation. No salary or compensation is paid to board members and/or committee volunteers for their contribution to the foundation. The majority of funds raised are given to research to improve the lives of those born with Krabbe disease.
Partners For Krabbe disease has been a member of Krabbe Translational Research Network (KTRN) for 8 years. For more information about the KTRN and the work they do, please visit:
...because we're in this together
Why Translational Research is important:
The last few decades have seen an explosion of scientific and technological breakthroughs. Unfortunately, patient care has not kept up with the rapid pace of new discoveries. The goal of translational research is to break down barriers between disciplines to quickly translate basic research discoveries into practical applications. This "bench-to-beside" approach requires basic scientists and clinicians to work more closely, with information exchanged in both directions. Community input keeps the research focused on issues that matter most to patients and their families.
P4KR supports the NDRD Brain and Tissue Bank at the University of Pittsburgh Medical Center in Pittsburgh PA. The NDRD Brain and Tissue bank was started in 2015 as an initiative with Partners For Krabbe Research to further research on post-mortem tissues from affected and transplanted patients with Krabbe disease.
Anne Rugari, founder of P4KR serves as the Consultant for the NDRD Brain and Tissue Bank. She coordinates the logistics involved between pathologists, clinicians, funeral homes, hospice, other social services when a family wishes to make a loved one’s tissue donation for research. As consultant, Anne is also involved with coordinating requests made by scientists and researchers who wish to obtain tissues for research from the NDRD Brain and Tissue Bank. Much has been discovered from the postmortem tissue samples of Krabbe patients. Partners For Krabbe Research is acknowledged in medical publications and journals for their work with the NDRD Brain and Tissue Bank.
Partners For Krabbe Research has also been acknowledged in a number of medical publications for their support in research. Here is the latest journal P4KR appeared in:
Molecular Therapy, The Society of Gene and Cell Therapy, entitled:
Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe’s disease after Global Gene Therapy.
Michael S. Marshall, et al 2018
Additional Services Provided by P4KR:
Funding of the NDRD Brain and Tissue Bank
Funding of equipment for research in animal models (UIC)
Supporter of KrabbeConnect : www.krabbeconnect.org
Demographic Study: P4KR is collecting information on Krabbe affected patients in order to have an accurate account of how many people are affected by Krabbe Disease all over the world. The information will help with advocating for therapy development, recruitment of clinical trials and will be used to support research to develop new treatments. The demographic study will be a support tool to improve the quality of lives for all who have encountered Krabbe Disease.
Please visit the Demographic Study Link on our website for more information or to participate in the study.
Other Organizations/Foundations, etc…
P4KR is involved with:
NORD (National Organization For Rare Disease)
Hunter’s Hope Family and Medical Symposium
Outreach and support to families who dealing with Krabbe Disease
Paul Fernhoff Lecture Series, Emory University
Guidestar Bronze Participant for non-profit transparency