This article and other news can be found in the latest issue of our newsletter which can be downloaded/viewed here: February 2021.
In May, BPANready turned 2 years old! It has been the fastest enrolling of our natural history studies. We broke 100 participants in last February 2020! We have also received 69 research blood samples and there have been nearly 400 study visits. None of this would have been possible without the BPAN family community’s enthusiastic research participation, and the grant funding we received from the NBIA Disorders Association and the University of Pennsylvania’s Orphan Disease Center Million Dollar Bike Ride. While the number of participants is important for the strength of a natural history study, so too is the number of years of data for each participant. We encourage everyone enrolled to keep inputting their data! We know that it can seem repetitive, but that is exactly what we need so we can learn how BPAN changes over time. Only with that background knowledge will we be able to tell if future therapeutics are changing the course of the disease. We need to be “BPAN ready” for treatment trials! We are currently exploring how to fund the BPANready study going forward. Our preliminary data analysis from the first two years has told us we are on the right track with our approach, but we need to continue to collect your family member’s data and samples over time.
The not-so-good news is that we are facing a funding crisis for BPANready. While we have some funding support for the PKANready and PLANready natural history studies, without further funding for BPANready, we will need to curtail our efforts on the study. This would have a detrimental impact on this important work, and while we are working hard to sustain this work, we need the family community’s support and funding. We have been exploring how to fund the BPANready study going forward, and will continue to do so. If families want to donate, they can do so here: Click HERE to Donate.