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 fundraising raised by 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 provided the NBIA Disorders Association with $4,867 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 community 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 provided its share of support 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 a joint grant 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 fundraising raised by 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 provided the NBIA Disorders Association with $5,000 from the German MDBR fundraiser.

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 fundraising raised by the Million Dollar Bike Ride 2020 resulted in a total grant from the University of Pennsylvania and the NBIA Disorders Association of $71,471. Hoffnungsbaum e.V. has provided the NBIA Disorders Association with € 2,217 from the German MDBR fundraising campaign.

Investigating C19orf12 functions in lipid metabolism
Arcangela Iuso, Institute of Neurogenomics at Helmholtz-Zentrum München, Germany

Thanks to the fundraising campaigns of Helf-Maya could Hoffnungsbaum 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 grant with 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 grant with 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 grant with NBIA sister organizations AISNAF (Italy) and NBIA Disorders Association (USA).

International NBIA Patient Registry, located at the Ludwig-Maximilians-University Munich (LMU)
coordinated by Thomas Klopstock, Friedrich Baur Institute, Munich, Germany

Like the NBIA Biobank at the Technical University of Munich, the International NBIA Patient Registry was established at LMU Munich in 2011-2015 as part of the EU-funded FP7 project TIRCON (Treat Iron-Related Childhood-Onset Neurodegeneration). The promotion of 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 critical NBIA research infrastructure. Financial support from the member organisations of the NBIA Alliance for Patient Registries and Biobanks 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 a joint grant with the NBIA's sister organizations AISNAF (Italy) and NBIA Disorders Association (USA).

Stimulation of the globus pallidus internus in patients with NBIA (formerly Hallervorden-
Sparrow syndrome): Prospective analysis of international therapy outcomes and development
of a therapeutic algorithm
Lars Timmermann, Amande Pauls, University of Cologne, Germany

Stimulation of the Globe 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

Total cost $30,000. The project was funded by the Research Fund of the NBIA Disorders Association, El Cajon, CA, USA, where Hoffnungsbaum e.V. paid out his share of $10,000 back in 2004.

BPAN Landscape Analysis
Science Compass, Milan (Italy)

Total cost of the analysis: € 43,920; The order is a cooperation between Hoffnungsbaum e.V. with the NBIA's sister organisations NBIA Disorders Association (US), AISNAF (Italy), Stichting Ijzersterk (Netherlands) and the BPAN patient organisations BPAN Warriors (US), BPAN France.

Preparation of an analysis of the medical research landscape with the aim of recording the state of the art in the field of BPAN, identifying research gaps and opportunities. In addition, he organized and moderated 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 cost of the analysis: € 23,228; The order is a cooperation between Hoffnungsbaum e.V. with the NBIA's 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 and research gaps and opportunities, as well as preparing and leading a scientific and medical advisory board to discuss priority research questions and develop a research strategy.