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.

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

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.

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.