March, 2018
The International NBIA Patient Registry and Biobank Needs Your Support!
The NBIA Alliance is asking for donations to the International Registry and Biobank during the month of March, while celebrating for the first time “NBIA Awareness Month” around the world. We hope to draw greater attention to Neurodegeneration with Brain Iron Accumulation (NBIA) disorders, and make NBIA Awareness a yearly event each March.
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 is from Retrophin Inc., a San Diego-based company that has begun testing a drug for PKAN patients. The registry and biobank are 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, once a steering committee made up of researchers, biotech, pharma and patient advocacy representatives that oversees the registry approves.
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 now Retrophin’s donation will carry the work through 2018, giving Alliance members and the NBIA community time to secure additional funding to sustain these valuable resources.
Please help us preserve and improve this vital resource to help find cures for all NBIA disorders.