Association chronicle

Focal points and milestones of the association's work from Hoffnungsbaum e.V. since 2002

After 17 years of association history, Hoffnungsbaum 2019 has a total of 91 association members representing 48 affected families. There were or are contacts to about 150 more affected families outside our association, many of them also abroad. With support from Hoffnungsbaum , the University Hospital of Munich was able to establish itself as the only clinical center with special NBIA expertise in Germany, and the network of contacts from Hoffnungsbaum to other hospitals and physicians with NBIA experience is steadily expanding. 72 diagnostic and counseling folders have been distributed to German-speaking NBIA families since 2012, often shortly after diagnosis. We have been able to produce and distribute 16 newsletters and 2 special supplements. We have supported the emergence of other NBIA patient organizations in other countries in an advisory capacity and significantly manage the affairs of the NBIA Alliance in cooperation with the NBIA Disorders Asscociation. From 2011 to 2015, we were a partner in an international research project funded by the European Union with our own remit. All this and the focal points and milestones of our work listed below are only the tip of the iceberg that we work through on a daily basis - while at the same time being affected families ourselves. We thank all those who support and accompany us in this process.



  • Organization of an event on Rare Disease Day in Heidelberg with a lecture on NBIA by the Second Chairman of Hoffnungsbaum

  • HoBa info table and presentation about Hoffnungsbaum by a board member at the Rare Disease Action Day in Ulm, Germany

  • First strategy meeting of the board of Hoffnungsbaum in Heidelberg and founding of a fundraising working group and a research working group

  • Second joint call for research grants from AISNAF, Hoffnungsbaum and NBIA Disorders Association.

  • Awarded a research grant to Northwestern University, Chicago, USA on the role of iron in the disease mechanisms of BPAN.

  • Participation of the Second Chairman of Hoffnungsbaum in a panel discussion at the Autophagy Congress in Tübingen, Germany.

  • Participation of the Second Chairman of Hoffnungsbaum in a German-American Workshop on Autophagy Diseases in New York and presentation of NBIA and Hoffnungsbaum there.

  • 3rd edition of the NBIA findings and consultation folder.

  • Publication of the new website of Hoffnungsbaum

  • Hoffnungsbaum Has the highest level of donations in its association history in 2019, thanks in particular to the private fundraising activities of two affected families with BPAN and MPAN


  • Hiring of a new part-time employee with reduced hours

  • Publication of a German translation of the PKAN treatment recommendations on the website of Hoffnungsbaum

  • Participation of HoBa members in Rare Disease Day events in Heidelberg and Ulm

  • First joint call for research grants by AISNAF, Hoffnungsbaum and NBIA Disorders Association.

  • Awarded 2 PKAN research grants to the University of North Carolina, USA, to develop a gene therapy and to the University of Brescia, Italy, to research a zebrafish model.

  • 8th NBIA Family Conference in Germany with 23 families (76 participants).

  • The new board of Hoffnungsbaum includes for the first time 2 relatives of BPAN patients

  • The implementation of the new General Data Protection Regulation requires a lot of time.


  • Creation and publication of a patient-oriented NBIA disease description on the Orphanet portal as part of the ACHSE project "Improving access to good patient information" and keynote presentation at a workshop on this project.

  • Contributed to the final version of the treatment recommendations for PKAN and publication of the article in the medical journal Molecular Genetics and Metabolism.

  • Participation of HoBa staff in the NBIA Scientific Conference in Stevenson (USA).

  • Financial participation of Hoffnungsbaum in a follow-up funding of the NBIA patient registry.

  • Since 2017, targeted fundraising for individual NBIA variants by affected families. The Milly's Mission fundraiser for BPAN research marks the beginning of this development.

  • Publication of a list of laboratories offering molecular genetic NBIA diagnostics in Germany on the NBIA Alliance website.


  • As part of the NBIA Alliance's follow-on funding of the NBIA International Patient Registry and Biobank, Hoffnungsbaum is making the first partial payment and organizing funding from the other Alliance partners for the Registry and Biobank

  • The Chair will speak at a satellite symposium at the annual meeting of the German Society for Pediatric and Adolescent Medicine (DGKJ) in Hamburg on "NBIA in Medical Care. Possibilities and challenges from the patient's perspective".

  • Revise and update the NBIA disease description on the Children's Network website.


  • Participation of a HoBa board member in the Rare Disease Day event in Hanover with an information table

  • Chair attendance at the European Congress of Neuropediatricians in Vienna to present a poster on the NBIA Alliance.

  • Information table about NBIA, Hoffnungsbaum e.V. and TIRCON at the congress of the German Society for Neurology in Düsseldorf

  • Co-organization of the TIRCON final conference in Munich and participation

  • Organization of the 4th NBIA Alliance meeting in Munich as part of the TIRCON final conference. The NBIA Alliance decides on a follow-up funding of the International NBIA Patient Registry and Biobank after the end of the EU project TIRCON.

  • Participation in the ACHSE seminar "International Networking" in Berlin and presentation of the NBIA Alliance in a lecture by a board member


  • Participation in the European Conference on Rare Diseases in Berlin with presentation of a poster introducing the NBIA Alliance.

  • Organization of the 3rd meeting of the NBIA Alliance in Berlin

  • 7th NBIA Family Conference in Germany with 15 families (69 participants).

  • Co-preparation and participation in the 3rd Scientific Symposium on Neuroacanthocytosis and NBIA in Stresa (Italy).

  • Hoffnungsbaum finances and organizes, within the framework of the NBIA Alliance, the awarding of travel grants to young scientists for their participation in the symposium in Stresa with

  • Hoffnungsbaum receives the "Hertie Prize for Commitment and Self-Help 2014


  • Presentation by the chair and HoBa info table at a symposium on rare neurodegenerative diseases at the annual meeting of the Society for Neuropediatrics in Innsbruck.

  • Dissemination of information on TIRCON clinical projects in patient-oriented brochures.

  • Lecture of the chairwoman at the annual meeting of the children's network in Aschaffenburg about networking and cooperation of Hoffnungsbaum

  • Participation in the content development of the TIRCON patient registry

  • Participation in TIRCON conferences in Munich and Warsaw and monthly telephone conferences of the steering committee


  • Opening event of TIRCON in Munich. Hoffnungsbaum leads the information dissemination working group in the project and is a member of the TIRCON steering committee.

  • Founding of the NBIA Alliance as an informal umbrella organization of NBIA patient organizations on the sidelines of the TIRCON meeting in Munich with NBIA patient representatives from 6 countries.

  • Hiring of a half-time employee as project staff for TIRCON and assistant to the Executive Board

  • Presentation of Hoffnungsbaum and TIRCON on the occasion of the awarding of the Eva Luise Köhler Prize for Research into Rare Diseases, an event organized by ACHSE e.V., in Berlin.

  • 6th NBIA Family Conference in Germany with 14 families from 4 countries (69 participants),

  • Introduction of a NBIA findings and advice folder as a service for affected families.

  • Hoffnungsbaum sponsors the 2nd Scientific Symposium on Neuroacanthocytosis and NBIA in Ede, The Netherlands. The chairwoman gives a presentation on the role of patient organizations in EU-funded research projects, using TIRCON as an example.

  • Hoffnungsbaum e.V. organizes the second meeting of the NBIA Alliance in Ede, NL

  • Participation of the Chair at the EURORDIS meeting of European rare disease patient organizations in Paris and presentation of the role of Hope Tree at TIRCON.

  • Own article about NBIA and TIRCON in "Dystonie aktuell", (German Dystonia Society


  • Hoffnungsbaum receives the Children's Network Award for "Good Cooperation".

  • Through Hoffnungsbaum , a neuropediatrician from the "Haunerschen Kinderspital" in Munich will speak about NBIA disorders at the annual conference of the German Society for Pediatrics and Adolescent Medicine (DGKJ) in Bielefeld.

  • Hoffnungsbaum e.V., AISNAF and NBIA Disorders Association are jointly funding the development of treatment recommendations for PKAN at the University of Oregon.

  • The TIRCON consortium is awarded the EU contract for a four-year international NBIA project.


  • Publication of the technical article on the retrospective study on the efficacy of deep brain stimulation in NBIA in the journal "BRAIN".

  • Chair participation in the inaugural meeting of the international neuroacanthocytosis (NA) research project EMINA in Munich, Germany.

  • 5th family conference in Germany with 62 participants (15 families from 4 European countries) and collection of blood samples for a project on neuroacanthocytosis/PKAN.

  • Participation of the chairwoman in a meeting of Munich NBIA and NA researchers with NBIA experts from the University of Oregon (USA) in Munich, the nucleus of TIRCON.

  • Application for EU funding by the NBIA research consortium TIRCON with 13 partners from 8 countries. Hoffnungsbaum e.V. and the NBIA Disorders Association are part of the consortium as full partners.

  • Chairwoman speaks for HoBa at MEDICA 2010 on cooperation between medical professionals and patient self-help


  • Travel grant from Hoffnungsbaum for a neurologist from the University Hospital of Cologne, Germany, a collaborator in the deep brain stimulation study we are sponsoring, to attend the 5th NBIA Family Conference in the U.S.

  • Financial support for a further study on deep brain stimulation in NBIA at the University Hospital of Cologne.

  • First international teleconference of the three NBIA patient organizations in the U.S. (NBIA Disorders Association), Germany (HoBa), and Italy (AISNAF) to explore future opportunities for collaboration

  • The Chair speaks about HoBa research funding at a NBIA Scientific Symposium in Munich on the occasion of the discovery of a new NBIA gene.


  • Participation of the chairperson at the central event for the first "Rare Disease Day" in Berlin

  • 4th NBIA family conference with 12 affected families (52 participants).

  • As part of the conference, study by a neurologist at the University Hospital of Göttingen on ultrasound diagnosis of NBIA, and conference participation by neuroacanthocytosis (NA) researchers (PKAN is also considered NA).

  • Publication of the new HoBa website

  • Hoffnungsbaum employs a part-time employee as part of a mini-job for the first time.


  • Online articles about life with NBIA and Hoffnungsbaum e.V. in the EURORDIS newsletter

  • Travel grant from Hoffnungsbaum for the principal investigator of the deep brain stimulation study we are sponsoring to attend the 4th NBIA Family Conference in the USA.

  • For his study on the efficacy of deep brain stimulation in NBIA, Prof. Dr. Timmermann of the University Hospital of Cologne receives the Klüh Foundation Award "for the Promotion of Innovation in Research and Science in the Field of Rare Diseases".

  • Lecture at an event of the health policy working group of the CDU NRW about self-help work for rare diseases based on the work of Hoffnungsbaum e.V.


  • Participation of the chairperson in the Mitochondrial Diseases Patient Day in Munich, personal meeting with physicians and scientists from the two Munich universities who are researching or showed interest in NBIA diseases.

  • Formation of the Medical Advisory Board for Hoffnungsbaum e.V.

  • Presentation of Hoffnungsbaum e.V. at the AOK symposium on rare diseases and publication of an article, "Networking is everything", in the G+G spezial 11/2006 of the AOK

  • 3rd family conference with 11 affected families (58 participants), collection of blood samples for the NBIA BioBank in the USA.

  • Funding of a retrospective multicenter study to investigate the efficacy of deep brain stimulation in NBIA patients at the Neurological University Hospital of Cologne, Germany.


  • Membership of HoBa in the Children's Network, ACHSE e.V. and EURORDIS

  • HoBa supports the participation of two German scientists at the 2nd NBIA Scientific Symposium in Gaithersburg in the U.S.

  • Presentation by the Chair about Hoffnungsbaum e.V. and its work at the 3rd NBIA Family Conference in the USA.

  • The Hoffnungsbaum chairwoman becomes a board member in the fall when ACHSE e.V. is founded with a focus on "Research in Rare Diseases" (until 2008).

  • 2nd NBIA family conference in Germany with 7 affected families (45 participants).


  • Participation in a symposium of the Children's Network on Rare Diseases in Munich, resulting in first contacts with the emerging "Alliance of Chronic Rare Diseases

  • Hoffnungsbaum receives its own logo and publishes its first club flyer

  • First NBIA family conference in Germany with 8 affected families

  • ZDF report in "Hallo Deutschland" about the family conference of Hoffnungsbaum

  • Hoffnungsbaum Provides a grant to an NBIA project at the University of Oregon, Portland, USA through the NBIA Disorders Association Research Fund.


  • 11.2.2003: Entry in the register of associations

  • Publication of own website

  • Publication of the own article "The Hallervorder-Spatz-Syndrome (HSS) - NBIA" in the medical journal "Kinderärztliche Praxis" under the heading "We help".

  • First Hoffnungsbaum-newsletter, which is published since then in loose succession

  • Start of the family network and the counselling for affected persons


  • On 24.11.2002 founding meeting with 3 affected families, a total of 19 members

  • 10.12.2002 Non-profit status

Subscribe to our Hoffnungsbaum newsletter!

With our newsletter emails you will receive valuable information about NBIA. If you would like to subscribe to additional info on individual NBIA variants or special info for researchers/clinicians besides the general newsletter, please click here: Subscribe additional info

Privacy policy

You have successfully registered!