The International NBIA Patient Registry and Biobank Needs Your Support!
The NBIA Alliance is asking for donations to the International Registry and Biobank to help with financial support.
The NBIA Alliance, an umbrella organization for NBIA patient advocacy groups around the world, received a $122,000 donation to sustain a critical research tool: the International NBIA Patient Registry and Biobank from July 2017 to December 2018. The gift was from Retrophin Inc., a San Diego-based company that was testing a drug for PKAN patients.
The registry and biobank are currently being used to advance NBIA research, enable faster access to worldwide clinical trials, and draw greater interest from potential funding sources to bring a drug to trial. It will assist researchers working on these diseases with the natural history studies data, which give insights into the course of a disease.
The data will be made available to other parties, including pharmaceutical companies, a steering committee made up of researchers, biotech, pharma, and patient advocacy representatives that oversee the registry approval.
All information in the registry is entered without any identifying information to protect the privacy of individuals. Only the clinician entering the data has access to the name of the patient. NBIA families participating in natural history studies with clinicians should inquire if they are also participating in the International NBIA Patient Registry to ensure their anonymized data is part of this important resource.
The registry and biobank resources were created by the NBIA research community to have a much-needed central repository of data. It was done under the European Union grant Treat Iron-Related Childhood-Onset Neurodegeneration (TIRCON.), which also spearheaded the first international NBIA clinical trial to test the drug deferiprone. TIRCON provided the funding for the registry and the biobank until the grant ended on Oct. 31, 2015.
In January 2016, the then-eight Alliance member organizations pledged a combined 84,400 euros for 18 months of funding for the registry and biobank. This covered costs through June 2017, and Retrophin’s donation carried the work through 2018, giving Alliance members and the NBIA community time to secure additional funding to sustain these valuable resources. The NBIA Alliance continues to cover the registry licensing fees of approximately 10,000 euros each year.
Please help us preserve and improve this vital resource to help find cures for all NBIA disorders.