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Radfahren für BPAN-Forschung – Million Dollar Bike Ride 2023
Auch in diesem Jahr sammelt der Million Dollar Bike Ride (MDBR) der University of Pennsylvania in Philadelphia Spenden für die Erforschung seltener Erkrankungen. Die 10. Ausgabe des Benefizradrennens wird am 10. Juni 2023...
Fundraising-Aktion des Lions-Club Mühlacker für Hoffnungsbaum e.V.
Mühlacker, Dezember 2022: Im Rahmen eines Club-Abends des Lions-Club Mühlacker hatte das Hoffnungsbaum e.V. Vorstandsmitglied Tiemo Durm die Möglichkeit einen Vortrag über den Verein sowie dessen Ziele und Hintergründe zu halten. Die Inhalte stießen beim Lions-Club...
International NBIA Symposium held in Switzerland in October 2022
2020 fand das letzte NBIA-Wissenschaftlertreffen pandemiebedingt nur online statt. Im Oktober 2022 trafen sich nun endlich zum ersten Mal seit 2017 zahlreiche Akteure in der NBIA-Forschung wieder persönlich für 3 Tage zum 8. Internationalen NBIA-Symposium in...
Care guidelines for PLAN are now in the works
Übersetzung eines englischen Artikels von Patricia Wood Dank der Unterstützung von vier Organisationen, die das Projekt finanzieren, sind nun Konsensleitlinien für die Behandlung und Versorgung bei einer Form von NBIA, die als PLAN oder PLA2G6-assoziierte...
INAD gene therapy moves one step closer
Gekürzte Übersetzung eines englischen Artikels von Patricia Wood Die Arbeit an einer Gentherapie für Infantile Neuroaxonale Dystrophie, genannt INAD, bekam im Oktober einen großen Anschub, als eine Londoner Biotech-Firma ihre Absicht bekundete, dabei zu helfen, die...
PKAN study with CoA-Z completed in the USA
Die Oregon Health & Science University in Portland (USA) hat von 2019 bis 2022 mit einem neuen Präparat namens „CoA-Z“ zur Behandlung von PKAN eine erste klinische Studie für PKAN-Patienten in den USA durchgeführt. Dieser erste Studienteil wurde inzwischen...
Two MPAN project grants totaling $140,000 awarded.
Hoffnungsbaum e.V. In partnership with three sister organizations in Europe and the United States, has awarded two MPAN grants to advance research priorities in mitochondrial membrane protein-associated neurodegeneration (MPAN) ...
Mulled wine stand in aid of Hoffnungsbaum and Team Bananenflanke
The Sandbox Friends, Melanie Schratzenstaller, Michael Bogner, Stephan Thalhammer, Stefan Huber, Ramona Klement and Lisa Kaltenecker, from Eberspoint (south of Landshut) organized a mulled wine stand again on the first weekend in December after a break of two years...
December 7, 2022, Online Lecture: The Gailinger Concept for Dealing with Dysphagia.
I was able to get to know the "Gailinger concept for dealing with swallowing disorders (not only) in children" during a rehabilitation stay of my son in the Hegau-Jugendwerk. Our son has benefited a lot from it and so I am happy that we have Christina Rauber (MSc and speech therapist)...
October 12, 2022, online lecture: Understanding hereditary developmental disorders better - what can be learned from the natural history of the disease.
Steffen Syrbe is a pediatric neurologist and professor of pediatric epileptology at Heidelberg University Hospital. He has been working with children with neurodevelopmental disorders for over 15 years. During this time, he has developed a strong interest in genetic causes of...
8th International Symposium on NBIA in Switzerland
NBIA scientists will meet to share their research Oct. 13-15 in Lausanne, Switzerland, as part of the 8th International NBIA Symposium. More than 20 scientists and physicians from around the world will present the results of their NBIA projects,...
Research project leads to a stem cell model of FAHN
A German team of scientists awarded a $45,000 research grant from the NBIA Disorders Association in 2020 has successfully produced a stem cell model of FAHN to help researchers better understand the disease and...
Pilot Grant of $69,775 announced for BPAN research.
The Million Dollar Bike Ride 2022 (Learn more about this fundraiser) raised $69,775, which has now been put out to bid by the Orphan Disease Center for a research project on BPAN. Researchers can apply for a pilot at the following link....
Cycling for BPAN - Million Dollar Bike Ride 2022 again a great success!
Video: NBIA DA It worked again - in 2022, the Million Dollar Bike Ride (MDBR, https://www.milliondollarbikeride.org/) at the University of Pennsylvania in Philadelphia once again far surpassed the US$20,000 mark: Nearly US$39,000 was raised....
NBIA Family Conference 2022 postponed!
Hoffnungsbaum e.V. wanted to organize the 9th NBIA Family Conference in Germany in September 2022 in the youth hostel of Heilbronn. We were very much looking forward to finally having a face-to-face meeting of NBIA families again after two years of the pandemic....
July 6, 2022, Online Lecture: Myofasciotomy - a minimally invasive treatment for shortened muscles.
NBIA disorders are often associated with restricted movement, changing muscle tension and involuntary spasms (dystonia), causing the muscles to become progressively shorter. Under certain conditions, myofasciotomy can provide some relief in this...
Press Release from CoA Therapeutics on Phase 1 Study for PKAN
We hereby issue the following press release from CoA Therapeutics: "CoA Therapeutics and BridgeBio Pharma present Phase 1 study data in healthy subjects to support the development of BBP-671 for PKAN and organic acidemias On May 26,...
CoA Therapeutics unveils timeline on path to PKAN clinical trial
CoA Therapeutics, a BridgeBio affiliate, has completed a Phase I study in healthy volunteers evaluating the safety, tolerability and drug-like properties of BBP-671. We present the data from healthy...
Survey helps prepare clinical trial for PKAN patients
California-based CoA Therapeutics is currently preparing a clinical trial of a new drug for PKAN patients. To make this study as patient-centered as possible, CoA Therapeutics developed a survey last year in which the...
German-British family takes up the fight with BPAN
Kelly Sayers and Oliver Feiler are parents of two children: Max (8 years old) and Emily (5 years old). The family lives near London, England. 1 ½ years ago, they received the devastating news that Emily had BPAN. BPAN...
Board member Noah Rusch gives interview on Rare Disease Day
Noah Rusch gives insights into the challenges and family life with his son Laurin, who suffers from BPAN, in an interview. The interview was published by the Outpatient Children's Hospice Foundation Munich for Rare Disease Day 2022:...
Million Dollar Bike Ride research grant awarded to BPAN.
The Center for Rare Diseases at the University of Pennsylvania, supported by the NBIA DA, has awarded a research grant to study the role of iron in the NBIA disease BPAN. The $66,366 awarded will be given to Young-Ah Seo of the...
Current call for an MPAN grant reflects clearly defined priorities
Hoffnungsbaum e.V. is partnering with three sister organizations on a current call for grants of up to $145,000 for one two-year or two one-year MPAN research projects:...
Call for proposals on MPAN research open by May 2, 2022
Associazione Italiana Sindromi Neurodegenerative da Accumulo di Ferro (AISNAF, Italy), Hoffnungsbaum e.V. (HoBa, Germany), NBIA Disorders Association (NBIADA, USA), and Stichting Ijzersterk (The Netherlands) are soliciting the submission of research projects by...
Generation change in the Management Board of Hoffnungsbaum
On Nov. 21, 2021, the annual membership meeting of. Hoffnungsbaum e.V. (HoBa) took place - with groundbreaking decisions for the future of our NBIA patient organization through the completion of the generational change in the association's leadership and the reorganization...
Guide with recommendations for the treatment of BPAN available
Researchers from various medical institutions around the world, with the support of affected families, recently published a guide for the treatment of beta-propeller protein-associated neurodegeneration (BPAN). The publication serves in...
New BPAN mouse model from Munich now available for researchers
With the publication of a long-awaited paper on a BPAN mouse model, researchers around the world now have access to these mice for studies. Scientists led by Dr. Holger Prokisch of the Technical University of Munich have developed a...
PKAN drug screening at Yale University.
A new one-year PKAN research project has been underway at Yale University, New Haven (USA) since July 2021 under the leadership of Professor Choukri Ben Mamoun. The project, entitled: "A High-Throughput Screening for PKAN-Revising Agents," will be conducted in the...
RARE-X: A new, secure database for exchanging BPAN health data.
BPAN sufferers and their families are invited to post health information about the disease on the new, secure RARE-X platform. This database is designed to collect information about rare diseases like BPAN and make it available to research to...
Million Dollar Bike Ride successful again with support from Hoffnungsbaum
Once again this year, the charity bike race at the University of Pennsylvania in Philadelphia in the USA, the Million Dollar Bike Ride 2021 was held virtually. It serves to raise funds for rare disease research, which this year was supported by 52...
Lena Burbulla discovers new possible BPAN disease mechanisms.
Prof. Dr. Lena Burbulla recently successfully completed a project investigating beta-propeller protein associated neurodegeneration (BPAN). The project, funded in 2019 with a total budget of €65,000 by Aisnaf, NBIADA and Hoffnungsbaum e.V. jointly...
News from CoA Therapeutics: Phase 1 trial with new drug for PKAN has started
There is recent news from CoA Therapeutics, Inc, a subsidiary of BridgeBio Pharma, Inc. The California-based company is working on a new potential therapy for PKAN patients. "CoA" stands for Coenzyme A and already in the company's name indicates...
NBIA Disorders Association family conference sessions now available online
The virtual conference platform is now open to all, tuition-free, so interested English-speaking families can view those sessions that are available as recordings. The program includes patient-focused research updates...
Cycling for Laurin as part of the Million Dollar Bike Ride
As part of the Million Dollar Bike Ride (MDBR), we started a fundraiser in our backyard at home in support of BPAN. Friends, relatives and acquaintances were invited to come by our house on their bikes and bring a donation. In return, there were fresh...
Cycling for BPAN - Million Dollar Bike Ride 2021
It's that time again: The Million Dollar Bike Ride (MDBR) of the University of Pennsylvania in Philadelphia will take place for the 8th time until June 12, 2021. Due to the pandemic, this charity bike race will once again take place not only in the USA, but virtually all over the...
PKAN family launches fundraising campaign - newspapers report
Shortly after their 11-year-old daughter Vivienne was diagnosed with PKAN last year, the Helmold family set out and began fundraising for the PKAN Fund at Hoffnungsbaum to benefit research into one of the most common forms of NBIA, because nothing...
First Online Lecture: 28 Participants at Lecture on BPAN Research in Tübingen
On February 10, 2021 Hoffnungsbaum e.V. launched a new online service offering for our families, especially in German-speaking countries. There will be a loose sequence of video meetings on various topics relevant to NBIA sufferers....
Review of TIRCON and its relevance to NBIA research and care.
On the topic of movement disorder networks, a retrospective article on TIRCON (Treat Iron-Related Childhood-Onset Neurodegeneration) has now appeared in the journal Frontiers in Neurology. From 2011 to 2015, TIRCON is a European Union-funded...
Researcher at Yale University works on new therapeutic approach for PKAN
From the USA we received the news that a potential new therapeutic option for PKAN is being researched at Yale University. This new approach is still at the very beginning of its development and a lot of scientific work and time is still needed to...
Hoffnungsbaum Info letter 2020
In 2020, we have again compiled an information letter for you. It contains information on the association's activities in 2020 and an outlook for next year. If you always want to be kept up to date, then subscribe to our online newsletter.
PKAN family launches another fundraising campaign
As at the beginning of the year, the Bianchi family is now once again actively collecting donations during Advent for the benefit of PKAN research, as their 3-year-old son Giulio suffers from this form of NBIA. In their hometown Kronau, parents Melanie and Tony have set up a booth with...
Record number of participants at the 7th International Scientific NBIA Symposium
Authors of the original English article: Patricia Wood and Angelika Klucken 160 participants from 26 countries attended the 7th International Symposium on NBIA and Related Disorders, held for the first time as a virtual event. Because online access made it...
Interim results of four BPAN research projects published
The NBIA Disorders Association, our sister organization in the U.S., reports in its fall newsletter on progress to date in four research projects on beta-propeller protein-associated neurodegeneration (BPAN). BPAN, one of the...
Results of the Hoffnungsbaum-survey 2020
In the period August - September 2020 Hoffnungsbaum e.V. conducted a broadband opinion survey among all association members as well as NBIA families in Germany, Austria and Switzerland with whom we are in contact. The aim of the survey was to explore...
Spontaneous donation campaign achieves more than 15.000 € for PKAN research
Last year, the Bianchi family received the diagnosis of PKAN for their son Giulio, who is now 3 years old. The parents then launched a fundraising campaign via Paypal and a Christmas card campaign in private for the benefit of PKAN research and are making...
Hoffnungsbaum e.V. finances MPAN project at Helmholtz Zentrum München
Hoffnungsbaum e.V. awards funding of € 151,540 to Dr. Arcangela Iuso and her team from the Institute of Neurogenomics at the Helmholtz Center in Munich. The grant will fund a 27-month research project to uncover essential...
Research team from Rostock receives grant for FAHN-research
Our sister organization in the U.S., the NBIA Disorders Association, has awarded a $45,000 grant to study the NBIA variant FAHN to a team of German scientists studying stem cells from patients with FAHN disease.....
Hoffnungsbaum and Team BPAN Germany contribute to the success of the Million Dollar Bike Ride
This year, the seventh edition of the charity bike race at the University of Pennsylvania in Philadelphia in the USA, the Million Dollar Bike Ride 2020, took place under difficult conditions. It serves to raise funds for research into rare...
NBIA Symposium 2020 takes place online
The 7th International Symposium on NBIA and Related Disorders will now be held virtually September 30 - October 3, 2020. Save the date for our virtual symposium! - Updates on new findings in NBIA diseases....
7th International NBIA Symposium in Lausanne postponed!
Postponed to Fall 2021 due to Corona situationUnfortunately, the 7th NBIA International Symposium will not take place in Lausanne, Switzerland, from October 1-3, 2020, and will be postponed to Fall 2021. For three days, scientists and physicians from...
Hoffnungsbaum Information letter at the end of 2019
Hoffnungsbaum e.V. sent this winter for the last time a newsletter as a print edition, which is available here as a download: Hoffnungsbaum-Infobrief-Winter-2019 In the future, we will mainly be communicating via our New Website or via our...
New research project on BPAN in Tübingen
Tübingen research group led by Professor Tassula Proikas-Cezanne and Hoffnungsbaum e.V. join forces in research on BPAN Deciphering the causes of rare, neurodegenerative diseases is a lengthy process that involves a lot of effort for those affected by...
PKAN clinical trial in USA and Canada to start in December 2019
For several years, the research teams of Dr. Susan Hayflick (University of Oregon, Portland, USA) and Prof. Ody Sibon (University of Groningen, NL) have been working on a new drug for PKAN and further deciphering the PKAN disease mechanisms. Now...
As Hoffnungsbaum representative at the Autophagy Congress in New York
Markus Nielbock was invited to a workshop in New York by Prof. Christian Behl, head of the Institute of Pathobiochemistry at the University of Mainz.
Autophagy Congress in Tübingen with panel discussion
On September 24, 2019, as the father of a daughter with BPAN, as well as in my capacity as Second Chair of Hoffnungsbaum e.V., participated in a public panel discussion on "Autophagy and Its Importance for Health and Neurodegeneration." They...
First results from FORT study for PKAN
The pharmaceutical company Retrophin had developed the drug Fosmetpantotenate for the treatment of PKAN. This was tested for safety and efficacy in a placebo-controlled and multicenter double-blind clinical study (FORT study)....
BPAN research project funded: Iron and neurodegeneration
In 2019, our Italian sister organization AISNAF again led an international call for BPAN research. This was concluded at the end of July with the funding of a new research project on BPAN. Thanks to the joint...
PKAN: results from clinical trial with deferiprone
From 2011 - 2015, the international NBIA research project TIRCON (Treat Iron-Related Childhood-Onset Neurodegeneration) was financially supported by the European Union. A major TIRCON subproject was a clinical trial to investigate the safety...
Benefit "Strong together for Maya" for MPAN research
In Lauterbach, a large charity event was held on June 1, 2019. The fate of 12-year-old Maya, who has the NBIA variant MPAN, moves the whole village, many other people in the Saarland and beyond. They all want to support Maya and...
MPAN can also be inherited in an autosomal dominant manner
Dr. Susan Hayflick's NBIA research group at Oregon Health & Science University in Portland, OR, USA, has found that mitochondrial membrane protein-associated neurodegeneration (MPAN) can also be inherited in an autosomal dominant manner. MPAN belongs...
Financing of a second PKAN project after the 2018 call for proposals
Dario Finazzi and his team at the University of Brescia are supported for their research on PKAN zebrafish by AISNAF, Hoffnungsbaum e.V. and NBIA Disorders Association support. As part of the 2018 joint international funding initiative, the research led by Lauriel Earley...
Call for proposals for funding NBIA research projects.
NBIA patient organizations AISNAF (Italy), Hoffnungsbaum e.V.,(Germany) and NBIA Disorders Association (USA) announce a joint funding initiative to award research grants to study the following two NBIA variants:...
Half marathon became BPAN fundraising run
Participants from MPIA at this year's SAS Half Marathon in Heidelberg, Germany, Image: MPIA/J. Henshaw On April 7, 2019, 14 colleagues of our board member Markus Nielbock from the Max Planck Institute for Astronomy (MPIA) made a...
Rhein-Neckar-Zeitung reports about Milly's mission
February 28 is International Rare Disease Day. Around the globe, numerous events have already been taking place for days and also today to mark the occasion. The interest of the media is also great. Rare Disease Day is intended to draw attention to the...
News from Maya and the MPAN fundraiser
In December, we presented the Matthiesen family's fundraising campaign in support of MPAN research. Maya herself actively supports this crowdfunding campaign launched by her parents shortly after her diagnosis. Together with her big brother, she...
Rare Disease Day in Heidelberg and Ulm 2019
February 28 is once again Rare Disease Day, which is celebrated all over the world. In cooperation with the Alliance of Chronic Rare Diseases - ACHSE e.V., the umbrella organization for rare diseases in Germany, events will be held to mark this...
Three NBIA patient organizations fund PKAN gene therapy project.
The first international call for research projects by AISNAF as part of a joint initiative with Hoffnungsbaum e.V. (Germany) and the NBIA Disorders Association (USA) has been completed: A project to develop a gene therapy for PKAN...
Matthiesen family launches fundraising campaign for MPAN research
The Saarland Broadcasting Corporation (SR) drew attention to the family's situation and their fundraising campaign in a sensitive television report on November 29 as part of its magazine "Wir im Saarland" (We in Saarland). Just the previous September, the Matthiesen family had been diagnosed with...
8th NBIA Family Conference in Germany 2018
On September 21-22, 2018, our 8th NBIA Family Conference took place in Düsseldorf with more than 75 participants. In addition to family members and affected individuals, the main participants included physicians, scientists, and a team of youth workers. Most of the guests...
News from Milly and her mission
On 22.12.2017 the Rhein-Neckar-Fernsehen (RNF), presented Milly and her family in the program RNFlife: http://www.rnf.de/mediathek/video/heidelberg-wie-die-kleine-emilia-gegen-ihre-gen-krankheit-ankaempft/ Emilia has BPAN. About a year ago, the parents...
Progress at NBIA Symposium & NBIA Alliance Meeting.
The latest research findings were presented at the 6th International Symposium on Neurodegeneration with Brain Iron Storage (NBIA) and Related Disorders. About 70 physicians, scientists and patient representatives from 12 countries came to...
TIRCON project ended with final conference in October 2015
In October 2015, the international research project TIRCON ("Treat Iron-Related Childhood-Onset Neurodegeneration"), which was funded by the European Union for four years, ended as planned. This event was celebrated with a major final conference on 15 and...
Hertie Foundation Commitment Award goes to Hoffnungsbaum e.V.
The non-profit Hertie Foundation has again awarded the Prize for Commitment and Self-Help. The prize honors exemplary activities of self-help groups and particularly committed people in the field of multiple sclerosis and neurodegenerative...
