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 collaborative 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 fundraising total generated 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. Provided $5, 000 to the NBIA Disorders Association from the MDBR German fundraiser 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 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. hasprovided € 2,217 to the NBIA Disorders Association from the German MDBR fundraiser.

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

Thanks to the fundraising efforts of Helf-Maya and was able to promote Hoffnungsbaum this project alone.

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 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 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 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 TU Munich, the International NBIA Patient Registry at LMU Munich was established 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 Alliance, to maintain and further develop the NBIA Patient Registry and Biobank as a critical NBIA research infrastructure. The financial support from the NBIA Patient Registry and Biobank Alliance member organizations totals € 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 collaborative funding effort 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 therapeutic outcomes 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. Hoffnungsbaum e.V. disbursed its share of $10,000 back in 2004.

Grant for the "8th International Symposium on NBIA and related Diseases"
Lausanne, October 13-15, 2022

The purpose of the grant is to network and support scientific exchange and the formation of collaborations among NBIA researchers.

BPAN Landscape Analysis
Science Compass, Milan (Italy)

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

Preparation of an analysis of the medical research landscape with the aim of mapping the state of the science in the field of BPAN, identifying research gaps and opportunities. In addition, organization and facilitation of a meeting of the Scientific and Medical Advisory Board (SMAB) to establish strategic priorities and goals for BPAN.

MPAN Landscape Analysis
Science Compass, Milan (Italy)

Total cost of the analysis: € 23,228; The assignment is a cooperation of Hoffnungsbaum e.V. with the NBIA sister organizations Aisnaf (Italy) and NBIA Disorders Association (USA)

Comprehensive study of the MPAN medical and scientific landscape, outlining the state of knowledge in the field and research gaps and opportunities, and prepare and lead a scientific and medical advisory board to discuss priority research questions and develop a research strategy.