As part of the Million Dollar Bike Ride 2022 (Further information on this fundraising campaign) raised $69,775, which has now been announced by the Orphan Disease Center for a research project on BPAN. Researchers can apply for a pilot grant at the following link: https://orphandiseasecenter.squarespace.com/grants/event-five-pk3yg-b7zlm-m4ag9
Video: NBIA DA
It worked again – in 2022, the Million Dollar Bike Ride (MDBR, https://www.milliondollarbikeride.org/) at the University of Pennsylvania in Philadelphia, the US$ 20,000 mark was exceeded by far: almost US$ 39,000 in donations were collected!
The interesting thing about the MDBR is that the donations collected are doubled by the University of Pennsylvania for a sum between US$ 20,000 and 30,000. As a result, the US$ 38,735 collected became around US$ 68.735, which can now be put out to tender again for a new BPAN research project.
This year, this charity bike race returned to its original mode as a live event in Philadelphia, USA. However, people from all over the world were once again able to participate virtually. This gave us in Germany the chance to be there again to collect donations for the NBIA variant BPAN together with our partner organization in the USA, the NBIA Disorders Association (NBIA DA). Hoffnungsbaum e.V. called for donations in Germany. So also called Noah Rusch He and his family went cycling again for his son Laurin and, like last year, invited friends, relatives and acquaintances to cycle to him, where the guests were treated to pizza from the wood-fired oven. In return, the guests brought plenty of donations for BPAN research. This campaign raised almost €2,980. But other families also collected donations and so another 1680 € were collected. Hoffnungsbaum e.V. was able to transfer the proud sum of 4,660 € (US$ 4,867) to the USA. We are very grateful to all donors, but also to the many helpers who have made this success possible!
Impressions from the MDBR 2022 in the USA and from the "Cycling for Laurin" campaign:
Photos: Noah Rusch
Kelly Sayers and Oliver Feiler are the parents of two children: Max (8 years old) and Emily (5 years old). The family lives near London, England. 1 1/2 years ago, they received the shocking news that Emily had contracted BPAN. BPAN (beta-propeller protein-associated neurogeneration) is currently the most common variant of NBIA (neurodegeneration with iron storage in the brain), accounting for about 40% of all diagnoses.
"After initial difficulties in dealing with the diagnosis, we started collecting donations 2 weeks ago and introducing this very rare disease to a wider audience," says Oliver Feiler. The family works closely with the children's hospital Great-Ormond-Street Hospital (GOSH) in London and the scientist and physician Prof. Manju Kurian. She is one of the world's leading pediatric neurologists and an expert on NBIA.
Kelly Sayers and Oliver Feiler have recently started asking for support from their friends and professional networks. To this end, they have founded the fundraising campaign "Emily Feiler's Fight": https://www.justgiving.com/fundraising/emilyfeilersfight
But they don't want to leave it at that, but organize several fundraising events every year to constantly collect more donations for research into the disease and to work towards a therapy. Among the ideas is the ascent of the three highest mountains in Great Britain as well as various marathons for BPAN. The family also has good contacts with celebrities from sports and society, who can act as multipliers for their cause.
Kelly and Oliver have already achieved success in this regard. The BBC will produce a short report on Emily and BPAN and broadcast it nationwide. Previous similar broadcasts have been able to help generate significant donations.
Photo: Kelly Sayers
Noah Rusch gives an insight into the challenges and family life with his son Laurin, who suffers from BPAN. The interview was published by the Ambulant Children's Hospice Foundation Munich on the occasion of Rare Disease Day 2022: https://www.kinderhospiz-muenchen.de/zum-tag-der-seltenen-erkrankungen-2022/
Photos: Noah Rusch
The Center for Rare Diseases at the University of Pennsylvania, supported by the NBIA DA, has awarded a research grant to study the role of iron in the NBIA disease BPAN. Young-Ah Seo of the University of Michigan will receive the $66,366 award:
https://www.orphandiseasecenter.med.upenn.edu/awarded-grants/iem8yjznwv916kubyg5h4vdsqbddem-ac78h
The funding came from the Million Dollar Bike Ride 2021, which was also supported by "Team BPAN Germany" with $5,000, as we reported here:
https://www.hoffnungsbaum.de/mdbr2021erfolg/
Researchers from various medical institutions around the world, with the support of affected families, recently published a guide for the treatment of beta-propeller protein-associated neurodegeneration (BPAN). The primary purpose of the publication is to support physicians in providing the best possible care for people with this most common form of NBIA today. The article is available online at: https://doi.org/10.1111/dmcn.14980
It contains recommendations for the evaluation and treatment of individuals with beta-propeller protein-associated neurodegeneration (BPAN), developed by experienced experts in the treatment of this disease. The guide covers the clinical manifestations and progression of the disease, imaging findings, epilepsy features and genetics. In addition, the article contains suggestions for doctors, ranging from the initial examination to the treatment of the disease throughout life.
Most doctors have free access to the full article. Therefore, we would like to encourage all families affected by BPAN to inform their treating physicians about it and to discuss the results with them. The manuscript of the treatment recommendations is currently not available to the general public free of charge. However, the main and most important contents were presented by Markus Nielbock, the chairman of Hoffnungsbaum e. V., inserted into the corresponding German Wikipedia article:
https://de.wikipedia.org/wiki/Beta-Propeller-Protein-assoziierte_Neurodegeneration
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