August 24, 2020

The 2020 Million Dollar Bike Ride Pilot Grant Program is now open! The MDBR Pilot Grant Program provides a one-year grant to support research related to a rare disease represented in the 2020 Million Dollar Bike Ride.

This Request for Applications (RFA) is open to the international community. All individuals holding a faculty‐level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation are eligible to respond to this RFA. Letters of Interest (LOIs) are due no later than Friday, September 18, 2020 by 8pm EST. Full proposals are by invitation only and due no later than Monday, October 26, 2020 by 8 pm EST. 

If questions, please contact Samantha Charleston, Assistant Director of Patient Outreach and Program Management, ODC at scharle@upenn.edu, or (215) 573-6822.

Team NBIA Disorders represented the NBIA Disorders Association at the seventh annual Million Dollar Bike Ride for rare disorders which was held virtually this year. Our Team raised $35,000 for BPAN research and $30,000 was matched by the University of Pennsylvania’s Orphan Disease Center Pilot Grant Program. We also received some sponsorship dollars that was divided among the teams to raise our final amount available for a research grant.

One grant of $71,471 will be awarded for Beta-propeller Protein-Associated Neurodegeneration (BPAN).

BPAN RFA INFORMATION

BPAN- A Neurodegeneration with Brain Iron Accumulation Disorder: One pilot grant for $71,471 is available for clinical and translational research studies related to the detection, diagnosis, or treatment of this rare, X-linked disorder caused by mutations in WDR45. BPAN typically is recognized in early childhood with delayed development and seizures. In adulthood, people with BPAN develop rapidly progressive parkinsonism. At the present time, symptoms may be treated but there are no cures.

Grants are expected to generate essential resources for the scientific community, advance knowledge about BPAN disease processes, and produce preliminary data to enable national and international funding to carry the work forward. Examples of priority topic areas include developing disease models that complement existing models, identifying biomarkers, delineating the molecular cascade that leads to early cellular changes, developing rational therapeutics, establishing outcome measures to be used in clinical trials, and developing other essential resources to substantially prepare the BPAN community for clinical trials. Natural history studies must have a component that includes participation in the International NBIA Patient Registry & Biobank. This grant is made possible by Team NBIA and BPAN families with the NBIA Disorders Association.

Please see 2020 MDBR RFA for more information.