Here you can see which research projects we have supported and how much we have donated. We finance our research funding primarily from donations from affected families, their relatives, friends and acquaintances, as well as from people and institutions affected by the fate of NBIA children, adolescents and adults.
Establishing autophagy inducers as novel therapies in cellular and animal models of Beta-propeller Protein-Associated Neurodegeneration (BPAN)
Bertrand Mollereau, Ecole Normale Supérieure Lyon (France)
The funds raised through the Million Dollar Bike Ride 2022 resulted in a total grant from the University of Pennsylvania and the NBIA Disorders Association of $69,775.00. Hoffnungsbaum e.V. has donated $4,867 to the NBIA Disorders Association from the German MDBR fundraiser in 2022.
Uncovering mitochondrial signature, dopamine metabolism and alpha-synuclein status in MPAN patient-derived dopaminergic neurons
Lena Burbulla, Ludwig-Maximilians-University, Biomedical Center, Munich, Germany
Elucidating the role of NBIA-mutated protein-c19orf12 in lipid homeostasis and mitochondrial dysfunction
Rajnish Bharadwaj, University of Rochester (USA)
The funding of the two projects resulted from a call for proposals totaling $140,000, which was a joint initiative of Hoffnungsbaum e.V. with the NBIA sister organizations, Aisnaf (Italy), NBIA Disorders Association (USA), Stichting Ijzersterk (Netherlands).
Best Practices in the Care and Management of People with PLAN
Susan Hayflick, Oregon Health & Science University (OHSU), Portland, OR, USA.
The total cost is $50,308.00. This grant is a joint funding of the PLAN patient organization INAD Cure Foundation (USA) with the NBIA sister organizations NBIA Disorders Association (USA), Aisnaf (Italy), Hoffnungsbaum e.V. has made its share of the funding available to the NBIA Disorders Association.
A High-Throughput Screen for PKAN Reversing Agents
Choukri Ben Mamoun, Yale University, School of Medicine, New Haven, CT, USA
Total cost: $115,000 This research grant was joint funding with NBIA sister organizations AISNAF (Italy) and NBIA Disorders Association (USA).
Reversing Brain Iron Overload in BPAN by a Natural Small Molecule
Young-Ah Seo, University of Michigan
The funds raised through the Million Dollar Bike Ride 2021 resulted in a total grant from the University of Pennsylvania and the NBIA Disorders Association of $66,366. Hoffnungsbaum e.V. has donated $5,000 from the German MDBR fundraiser to the NBIA Disorders Association.
Deciphering the causes of mitochondrial network disruption in WDR45-defective cells and their contribution to the BPAN pathology
Fulvio Reggiori, University Medical Center Groningen
The funds raised through the 2020 Million Dollar Bike Ride resulted in a total grant from the University of Pennsylvania and the NBIA Disorders Association of $71,471. Hoffnungsbaum e.V. has donated €2,217 to the NBIA Disorders Association from the German MDBR fundraising campaign.
Investigating C19orf12 functions in lipid metabolism
Arcangela Iuso, Institute of Neurogenomics at the Helmholtz Center Munich, Germany
Thanks to the fundraising campaigns of Helf-Maya Hoffnungsbaum was able to support this project on its own.
Mechanistic insights into iron accumulation in WDR45 mutant neurons linked to ß-propeller-associated neurodegeneration
Lena Burbulla, Northwestern University Chicago, IL, USA
Total cost: € 65,000 This research grant was a joint funding with the NBIA sister organizations AISNAF (Italy) and NBIA Disorders Association (USA).
Understanding and curing PKAN: advancements from the phenotypic rescue of a zebrafish model
Dario Finazzi, University of Brescia, Brescia, Italy
Total cost: € 22,000 This research grant was a joint funding with the NBIA sister organizations AISNAF (Italy) and NBIA Disorders Association (USA).
PANK2 gene therapy for treatment of PKAN and elucidation of disease biology
Lauriel Earley, University of North Carolina, Chapel Hill, USA
Total cost: € 39,500 This research grant was a joint funding with the NBIA sister organizations AISNAF (Italy) and NBIA Disorders Association (USA).
International NBIA Patient Registrybased at the Ludwig Maximilian University of Munich (LMU)
coordinated by Thomas KlopstockFriedrich Baur Institute, Munich, Germany
Like the NBIA biobank at the Technical University of Munich, the International NBIA Patient Registry at the LMU Munich was set up in 2011-2015 as part of the EU-funded FP7 project TIRCON (Treat Iron-Related Childhood-Onset Neurodegeneration). The funding from Hoffnungsbaum e.V. 2016 is part of a cooperative funding by the members of the NBIA Allianceto maintain and further develop the NBIA patient registry and biobank as a key NBIA research infrastructure. The financial support from the member organizations of the NBIA Alliance for Patient Registry and Biobank amounts to a total of € 84,400.
$ 16,116.67 (€ 12.395,95)
Best Practices in the Care and management of People with NBIA
Susan Hayflick, Oregon Health & Science University, Portland, OR, USA
Total cost: $48,350.01. This grant was joint funding with NBIA sister organizations AISNAF (Italy) and NBIA Disorders Association (USA).
Stimulation of the globus pallidus internus in patients with NBIA (formerly Hallervorden-
Spatz syndrome): Prospective analysis of international therapy results and development
of a therapeutic algorithm
Lars Timmermann, Amande Pauls, University of Cologne, Germany
Stimulation of the globus pallidus in NBIA: treatment results and development of a therapeutic algorithm
Lars Timmermann, University of Cologne, Germany
$ 10,000 (€7.872,56)
Dynamics in brain iron distribution in PKAN
Susan Hayflick, Oregon Health & Science University Portland OR, USA
The project was supported by the research fund of the NBIA Disorders Association, El Cajon, CA, USA, which paid its share of $ 10,000 in 2004. Hoffnungsbaum e.V. paid out its share of $10,000 in 2004.
Commissions and other subsidies
Hoffnungsbaum e.V. not only funds research projects, but also promotes measures designed to advance research.
BPAN Landscape Analysis
Science Compass, Milan (Italy)
Total costs of the analysis: € 43,920; The commission is a cooperation of Hoffnungsbaum e.V. with the NBIA sister organizations NBIA Disorders Association (US), AISNAF (Italy), Stichting Ijzersterk (Netherlands) and the BPAN patient organizations BPAN Warriors (US), BPAN France.
Preparation of an analysis of the medical research landscape with the aim of recording the state of science in the field of BPAN and identifying research gaps and opportunities. In addition, organization and moderation of a meeting of the Scientific and Medical Advisory Board (SMAB) to define strategic priorities and goals for BPAN.
MPAN Landscape Analysis
Science Compass, Milan (Italy)
Total costs of the analysis: € 23,228; The commission is a cooperation of Hoffnungsbaum e.V. with the NBIA sister organizations Aisnaf (Italy) and NBIA Disorders Association (USA)
Comprehensive study of the medical and scientific MPAN landscape, outlining the state of knowledge in the field, research gaps and opportunities, and preparation and management of a scientific and medical advisory board to discuss priority research questions and develop a research strategy.
Current calls for proposals and research funding
Here you can see tenders that Hoffnungsbaum e.V. has supported and find further information on some of the projects mentioned above. You can also see other research projects in which Hoffnungsbaum is involved.
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....
Mühlacker, December 2022: As part of a club evening organized by the Mühlacker Lions Club, board member Hoffnungsbaum e.V. Board member Tiemo Durm had the opportunity to give a presentation about the club and its aims and background. The content was well received by the Lions Club...
Hoffnungsbaum e.V. in conjunction with three sister organizations in Europe and the US, has awarded two MPAN grants to advance research priorities in the field of mitochondrial membrane protein-associated neurodegeneration (MPAN)...
The Sandbox Friends, Melanie Schratzenstaller, Michael Bogner, Stephan Thalhammer, Stefan Huber, Ramona Klement and Lisa Kaltenecker, from Eberspoint (south of Landshut) organized a mulled wine stand again on the first weekend of December after a break of two years...
The Million Dollar Bike Ride 2022 (more information on this fundraising campaign) raised $69,775, which the Orphan Disease Center is now offering for a research project on BPAN. Researchers can apply for a pilot project at the following link.
Video: NBIA DA It worked again - in 2022, the Million Dollar Bike Ride (MDBR, https://www.milliondollarbikeride.org/) of the University of Pennsylvania in Philadelphia was able to far exceed the US$ 20,000 mark: Almost US$ 39,000 was raised...
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. The announced $66,366 will be awarded to Young-Ah Seo of the...
Hoffnungsbaum e.V. is partnering with three sister organizations on a recent call for funding of up to USD 145,000 for one two-year or two one-year MPAN research projects:...
Associazione Italiana Sindromi Neurodegenerative da Accumulo di Ferro (AISNAF, Italy), Hoffnungsbaum e.V. (HoBa, Germany), NBIA Disorders Association (NBIADA, USA), and Stichting Ijzersterk (The Netherlands) are soliciting the submission of research projects by...
Since July 2021, a new one-year PKAN research project has been underway at Yale University, New Haven (USA) under the direction of Professor Choukri Ben Mamoun. The project, entitled: "A high-throughput screen for PKAN-revising agents", is being carried out as part of...
The Million Dollar Bike Ride 2021 charity bike race at the University of Pennsylvania in Philadelphia, USA, was once again held virtually this year. It serves to raise funds for research into rare diseases, which this year will be supported by 52...
The virtual conference platform is now open to all, free of charge, so that interested families with English language skills can watch those sessions that are available as recordings. The program includes patient-focused research updates...
Hoffnungsbaum e.V. awards funding in the amount of € 151,540 to Dr. Arcangela Iuso and her team from the Institute of Neurogenomics at the Helmholtz Centre in Munich. The funding will finance a 27-month research project aimed at uncovering the essential...
Our sister organization in the USA, the NBIA Disorders Association, has awarded a $45,000 grant for research into the NBIA variant FAHN to a team of German scientists studying stem cells from patients with FAHN disease....
This year, the seventh edition of the charity bike race of the University of Pennsylvania in Philadelphia in the USA, the Million Dollar Bike Ride 2020, took place under difficult conditions. Its purpose is to raise funds for research into rare diseases.
Tübingen research group led by Professor Tassula Proikas-Cezanne and Hoffnungsbaum e.V. join forces to research BPAN Deciphering the causes of rare, neurodegenerative diseases is a lengthy process that involves a great deal of effort for those...
Markus Nielbock was invited to a workshop in New York by Prof. Christian Behl, head of the Institute of Pathobiochemistry at the University of Mainz.
On September 24, 2019, as the father of a daughter with BPAN and in my role as Second Chairman of Hoffnungsbaum e.V.I took part in a public panel discussion on the topic of "Autophagy and its importance for health and neurodegeneration". You...
In 2019, our Italian sister organization AISNAF once again led an international call for proposals for BPAN research. This was concluded at the end of July with the funding of a new research project on BPAN. Thanks to the joint...
Dario Finazzi and his team at the University of Brescia are supported for their research on PKAN zebrafish by AISNAF, Hoffnungsbaum e.V. and NBIA Disorders Association for their research on PKAN zebrafish. As part of the 2018 joint international funding initiative, the project led by Lauriel Earley...
The NBIA patient organizations AISNAF (Italy), Hoffnungsbaum e.V.(Germany) and NBIA Disorders Association (USA) announce a joint funding initiative to award research grants for the study of the following two NBIA variants:...
The first international call for research projects by AISNAF as part of a joint initiative with Hoffnungsbaum e.V. (Germany) and the NBIA Disorders Association (USA) has been completed: A project to develop a gene therapy for PKAN...
The latest research findings were presented at the 6th International Symposium on Neurodegeneration with Brain Iron Accumulation (NBIA) and Related Diseases. Around 70 doctors, scientists and patient representatives from 12 countries came to...
In October 2015, the international research project TIRCON ("Treat Iron-Related Childhood-Onset Neurodegeneration"), which was funded by the European Union for four years, ended as planned. This event was celebrated with a major closing conference on 15 and...