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NBIA NEWS & INFORMATION

Team NBIA Disorders reaches $30,000 goal for MDBR

Matt and Josie BikingJune 2020

Team NBIA did it again! For the fourth consecutive year, the NBIA Disorders Association successfully met the goal set by the Million Dollar Bike Ride and will have $30,000 matched, dollar for dollar, by the University of Pennsylvania’s Orphan Disease Center.

The team’s efforts will result in a $60,000 grant for BPAN research to be awarded later this year. BPAN, which stands for Beta-propeller protein-associated neurodegeneration, has emerged as the most common NBIA disorder. The Million Dollar Bike Ride has been a big source of support for BPAN research, a priority of the NBIA Disorders Association, bringing in $130,000 in matching money alone from UPenn over the past three years. With this match, the BPAN grants will exceed $320,000.

Despite a global pandemic, the 7th Annual Million Dollar Bike Ride carried on. Virtually, of course. Originally set to take place in Philadelphia on June 13, this year’s ride was held in communities around the world to help rare disorders while maintaining social distancing amid COVID-19.

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2019 Million Dollar Bike Ride supporting BPAN stem cell project

Lockhart Australian familiesJune 2020

Thanks to money raised in last year’s Million Dollar Bike Ride, the NBIA Disorders Association is supporting a stem cell research project in Australia that will examine BPAN’s effects on the brain and drugs that could help treat the disorder.

Dr. Paul Lockhart of Murdoch Children’s Research Institute (MCRI) in Melbourne is leading the project and received a grant in February for $60,561 from the bike ride’s sponsoring organization, the University of Pennsylvania. Of that total, the NBIA community raised $30,561 and won the maximum match of $30,000 from the school’s Orphan Disease Center to study Beta-propeller Protein-Associated Neurodegeneration (BPAN).

Our organization was deeply involved in the grant-making process. We wrote the request for proposals, and members of our Scientific & Medical Advisory Board reviewed the applications and made recommendations. The University of Pennsylvania will manage the grant and send us a copy of the final scientific report that Lockhart’s team submits.

The project is titled “Development of novel human stem cell models of BPAN for disease modeling and drug screening,” and is being conducted by the Bruce Lefroy Centre, a genetics research unit at MCRI, where Lockhart is co-director. His co-investigators are Dr. Martin Delatycki and Dr. Jay Shukla.

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NBIA Disorders Association awards $45,000 for FAHN research in March

Luo Frech HermannJune 2020

The NBIA Disorders Association has awarded a $45,000 research grant to a team of German scientists studying stem cells in patients with the NBIA disorder known as FAHN.

Led by Dr. Andreas Hermann, along with Drs. Moritz Frech and Jiankai Luo of the University Medical Center Rostock, the team will create a model of FAHN, or Fatty Acid Hydroxylase-associated Neurodegeneration, in the lab, along with stem cells to better understand how the disease works. With that understanding, the researchers can advance to testing potential therapies to see whether they can reverse FAHN’s effects.

The team plans to create a supply of patient-specific induced pluripotent stem cells, which have the capacity to become any cell in the body. They can also self-renew, meaning that they divide and produce more stem cells.

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