Cycling for BPAN research - Million Dollar Bike Ride 2023

This year, the Million Dollar Bike Ride (MDBR) at the University of Pennsylvania in Philadelphia is once again raising funds for research into rare diseases. The 10th edition of the charity bike race will take place on June 10, 2023.

https://www.milliondollarbikeride.org/

Once again, it is possible to take part virtually. This gives us in Germany the chance to be part of it again. Be it just by collecting donations or by actually cycling along, having fun and at the same time drawing attention to NBIA and especially BPAN with photos.

This year, 39 different organizations are taking part and the NBIA variant BPAN will again be represented by our partner organization NBIA Disorders Association (NBIA DA) in the USA.

The interesting thing about the MDBR is that the donations collected are doubled by the University of Pennsylvania when they reach between US$ 20,000 and US$ 30,000. Last year, a research grant for BPAN in the amount of $69,755 was awarded to Professor Bertrand Mollereau at ENS-Lyon in France. Our goal is to exceed the US$ 20,000 mark again in 2023 together with the NBIA DA in order to initiate another new research project!

You can join in virtually as a participant and document your activities or simply donate. Officially, however, every active participant must register. However, virtual participation is free of charge. Please register here: https://www.milliondollarbikeride.org/2023-cyclist-registration

Markus Nielbock has once again created a donation page: https://charity.pledgeit.org/f/Cj497fpqdR You can use this page to donate directly to the organizers of the MDBR for BPAN research by credit card.

Impressions from last year, video: NBIA DA

However, as in previous years, you can also transfer your donations to Hoffnungsbaum . We collect these funds as "Team BPAN Germany" and then transfer them to our partners in the USA. In this way, you will receive a tax receipt for your donation. Please enter "MBDR 2023" or "Team BPAN Germany" as the intended purpose. The best way to do this is to use our online donation form: https://www.hoffnungsbaum.de/spenden/

You are all invited to become part of this team with friends, relatives, acquaintances, celebrities, athletes, colleagues, sponsors, ... and to cycle and collect donations for BPAN research. Encourage people around you to take part. Start campaigns in your area and draw attention to BPAN.

Post photos and videos on social media to raise awareness of the campaign using the hashtags: #MDBR2023 and #TeamBPANGermany or on Twitter and Instagram @MDBRide4Rare and on Facebook @milliondollarbikeride. Use our in photos and videos Team BPAN Germany Poster. You can also send us pictures for publication on this page or on our team website.

You are welcome to document your routes via the MDBR's STRAVA portal: https://www.milliondollarbikeride.org/strava

We are very confident that with your support and together with the NBIA DA we will surpass the US$ 20,000 mark and once again double the amount donated.

The contact person for "Team BPAN Germany" of MDBR 2023 is Markus Nielbock: markus.nielbock@hoffnungsbaum.de

German-British family takes up the fight with BPAN

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German-British family takes up the fight with BPAN

Kelly Sayers and Oliver Feiler are parents of two children: Max (8 years) and Emily (5 years). The family lives near London, England. 1 ½ years ago, they received the devastating news that Emily had BPAN. BPAN (beta propeller protein-associated neurogeneration) is currently the most common variant of NBIA (neurodegeneration with iron storage in the brain), accounting for around 40% of all diagnoses.

"After initial difficulties in dealing with the diagnosis, two weeks ago we started collecting donations and introducing this very rare disease to a wider audience," says Oliver Feiler. The family is working closely with 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 in NBIA.

Kelly Sayers and Oliver Feiler have recently started asking for support from their friends and professional networks. To this end, they have set up the "Emily Feiler's Fight" fundraising campaign: https://www.justgiving.com/fundraising/emilyfeilersfight

But they don't want to stop there, they want to organize several fundraising events every year to constantly collect more donations for research into the disease and work towards a therapy. Ideas include climbing the three highest mountains in the UK and various marathons for BPAN. The family also has good contacts with celebrities from sport and society who can act as multipliers for their cause.

Kelly and Oliver have already achieved one success in this respect. The BBC will produce a short report about Emily and BPAN and broadcast it nationally. Previous similar programs have helped to generate significant donations.

Photo: Kelly Sayers

Guideline with recommendations for the treatment of BPAN available

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Guideline with recommendations for the treatment of BPAN available

Researchers from various medical institutions around the world, with the support of affected families, have recently published guidelines for the treatment of beta propeller protein-associated neurodegeneration (BPAN). The main purpose of the publication is to support doctors 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) that have been developed by experienced experts in the management of this disease. The guide covers the clinical manifestations and progression of the disease, imaging findings, epilepsy characteristics and genetics. In addition, the article contains suggestions for physicians, ranging from the initial examination to the treatment of the disease throughout life.

Most doctors have free access to the full article. We would therefore like to encourage all families affected by BPAN to inform their treating physicians and discuss the results with them. The manuscript of the treatment recommendations is currently not available to the general public free of charge. However, Markus Nielbock, Chairman of Hoffnungsbaum e. V., has added the essential and most important content to the corresponding German-language Wikipedia article:

https://de.wikipedia.org/wiki/Beta-Propeller-Protein-assoziierte_Neurodegeneration

RARE-X: A new, secure database for the exchange of BPAN health data

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RARE-X: A new, secure database for the exchange of BPAN health data

BPAN sufferers and their families are invited to post health information about the disease on the new, secure RARE-X platform. The purpose of this database is to collect information about rare diseases such as BPAN and make it available for research. By linking data sets from different diseases, researchers can gain new insights that can be of decisive benefit in the development of therapies.

Launched on 6 August, the decentralized online data platform RARE-X-BPAN aims to promote data sharing and thus accelerate the dissemination of information and the pace of BPAN research. The data is collected and stored in a decentralized manner using standardized questions and is only brought together for analysis.

The platform is free for families to use. Importantly, it keeps health information confidential by only providing anonymized data, i.e. not linked to individual names. Individuals and families affected by beta propeller protein-associated neurodegeneration (BPAN) decide for themselves whether to allow or deny access to their personal health data for a research project.

The platform is currently open to English-speaking BPAN families. Those wishing to participate can access the website at: https://bpan.rare-x.org
A translation is planned for the coming year.

Six patient organizations representing people with BPAN (NBIA Disorders Association in the USA, Hoffnungsbaum e. V. in Germany, Stichting Ijzersterk in the Netherlands and three organizations that deal exclusively with BPAN: BPAN Warriors in the USA, BPAN France and Autour du BPAN, both in France) have made a start with RARE-X, a newly founded non-profit organization. Other rare diseases will gradually be included.

Additional information on the RARE-X database, its purpose and how it works is summarized in an online presentation, which you can access at:
https://www.youtube.com/watch?v=_QOZsX1SKOw The English-language presentation has subtitles that can be automatically translated into German.

Further information on the non-profit organization RARE-X can be found at: https://rare-x.org

Original article by Patricia Wood, translated and revised by Markus Nielbock

Million Dollar Bike Ride again successful with support from Hoffnungsbaum

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Million Dollar Bike Ride again successful with support from Hoffnungsbaum

This year, the charity bike race of the University of Pennsylvania in Philadelphia in the USA, the Million Dollar Bike Ride 2021 was held virtually. It serves to collect donations for research into rare diseases, which were represented by 52 organizations this year. The NBIA variant BPAN was organized for the fifth time by our partner organization NBIA Disorders Association (NBIA DA) in the USA. This eighth edition of the cycling campaign has raised a total of almost US$ 450,000 for BPAN research in recent years.

Normally, this event is centered around a bike ride in Philadelphia, but due to the Covid 19 pandemic, this was once again not possible. Instead, anyone around the world could take part at home. To this end, Markus Nielbock, Second Chairman of Hoffnungsbaum e.V.founded Team BPAN Germany back in 2020.

The goal of the Million Dollar Bike Ride was to raise between US$ 20,000 and US$ 30,000 with all participants by the end of June 2021. The University of Pennsylvania will double any amount raised within this range through sponsorship. The hoped-for US$ 60,000 would make it possible to fund a research project.

This year's proceeds of US$ 5,000 raised by Team BPAN Germany significantly exceeded last year's result. A large part of this amount was raised through the private fundraising event "Cycling for Laurin" by our Board member Noah Rusch . This success was additionally crowned by the fact that the hoped-for total sum of US$ 30,000 was once again exceeded. Therefore, a scholarship of more than US$ 62,000 can now be financed. Team BPAN Germany would like to thank all supporters for their participation in this campaign.

In the coming weeks, the Center for Rare Diseases at the University of Pennsylvania, supported by the NBIA DA, will announce a research grant for which physicians and researchers worldwide can apply. A jury of experts and patient representatives is expected to select the best project proposal in October. We will also inform you about this.