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 total amount of donations 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. provided the NBIA Disorders Association with $4,867 from the German MDBR fundraising campaign in 2022 for this purpose.

Uncovering mitochondrial signature, dopamine metabolism and alpha-synuclein status in MPAN patient-derived dopaminergic neurons
Lena Burbulla, Ludwig-Maximilians-Universität, 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 for 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 costs amount to $ 50,308.00. This funding is a joint financing project between the PLAN patient organization INAD Cure Foundation (USA) and the NBIA sister organizations NBIA Disorders Association (USA), Aisnaf (Italy). Hoffnungsbaum e.V. has made its contribution to 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 costs: $ 115,000. This research grant was a joint financing project with the 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 total amount of donations 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. provided the NBIA Disorders Association with $ 5,000 from the German MDBR fundraising campaign for this purpose.

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 total amount of donations raised through 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. provided the NBIA Disorders Association with € 2,217 from the German MDBR fundraising campaign for this purpose.

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 fund this project alone.

Mechanistic insights into iron accumulation in WDR45 mutant neurons linked to β-propeller-associated neurodegeneration
Lena Burbulla, Northwestern University Chicago, IL, USA

Total costs: € 65,000. This research grant was a joint financing project 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 costs: € 22,000. This research grant was a joint financing project 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 costs: € 39,500. This research grant was a joint funding initiative with the NBIA sister organizations AISNAF (Italy) and NBIA Disorders Association (USA).

International NBIA Patient Registry, located at the Ludwig Maximilian University of 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 at the LMU Munich was established from 2011-2015 as part of the EU-funded FP7 project TIRCON (Treat Iron-Related Childhood-Onset Neurodegeneration). The funding from Hoffnungsbaum e.V. in 2016 is part of a cooperative financing by the members of the NBIA Alliance, to maintain and further develop the NBIA Patient Registry and Biobank as crucial NBIA research infrastructure. The financial support from the member organizations of the NBIA Alliance for the 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 costs: $ 48,350.01. This grant was a joint funding initiative with the 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

Total costs $ 30,000. The project was funded by the research fund of the NBIA Disorders Association, El Cajon, CA, USA, to which Hoffnungsbaum e.V. paid its share of $ 10,000 back in 2004.

BPAN Landscape Analysis
Science Compass, Milan (Italy)

Total cost of the analysis: € 43,920; This project is a collaboration between Hoffnungsbaum e.V. and the NBIA sister organizations NBIA Disorders Association (US), AISNAF (Italy), Stichting Ijzersterk (Netherlands) and the BPAN patient organizations BPAN Warriors (US), BPAN France.

Creation 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. This includes organizing and moderating 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; This project is a collaboration between Hoffnungsbaum e.V. and the NBIA sister organizations Aisnaf (Italy) and NBIA Disorders Association (USA)

Comprehensive study of the medical and scientific MPAN landscape, presenting the state of knowledge in this field as well as research gaps and opportunities, as well as preparation and management of a scientific and medical advisory board to discuss priority research questions and develop a research strategy.