Latest posts
Here you will see the most recent posts from all categories.
Cycling for BPAN research - Million Dollar Bike Ride 2023
This year, the Million Dollar Bike Ride (MDBR) at the University of Pennsylvania in Philadelphia is once again raising funds for research into rare diseases. The 10th edition of the charity bike race will take place on June 10, 2023....
Fundraising campaign by the Mühlacker Lions Club for Hoffnungsbaum e.V.
Mühlacker, December 2022: As part of a club evening organized by the Mühlacker Lions Club, board member Hoffnungsbaum e.V. Board member Tiemo Durm had the opportunity to give a presentation about the club and its aims and background. The content was well received by the Lions Club...
International NBIA Symposium took place in Switzerland in October 2022
In 2020, the last NBIA scientists' meeting took place only online due to the pandemic. In October 2022, for the first time since 2017, numerous actors in NBIA research finally met in person again for 3 days for the 8th International NBIA Symposium in...
Care guidelines for PLAN are now in progress
Thanks to the support of four organisations funding the project, consensus guidelines for treatment and care in a form of NBIA known as PLAN or PLA2G6-associated guidelines are now available.
INAD gene therapy is one step closer
Work on a gene therapy for infantile neuroaxonal dystrophy, called INAD, got a big boost in October when a London-based biotech firm announced its intention to help improve the...
PKAN study with CoA-Z completed in the USA
From 2019 to 2022, Oregon Health & Science University in Portland (USA) conducted a first clinical trial for PKAN patients in the USA with a new drug called "CoA-Z" for the treatment of PKAN. This first part of the study has now been...
Two MPAN project grants totaling $140,000 awarded
Hoffnungsbaum e.V. in conjunction with three sister organizations in Europe and the US, has awarded two MPAN grants to advance research priorities in the field of mitochondrial membrane protein-associated neurodegeneration (MPAN)...
Mulled wine stand for the benefit 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 of December after a break of two years...
December 7, 2022, online lecture: The Gailinger concept for dealing with swallowing disorders
I got to know the "Gailinger concept for dealing with swallowing disorders (not only) in children" during my son's rehabilitation stay at the Hegau-Jugendwerk. Our son benefited greatly from it and so I'm delighted that we were able to introduce Christina Rauber (MSc and speech therapist)...
October 12, 2022, online lecture: Understanding hereditary developmental disorders better - what you can learn from the natural course 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...
8th International Symposium on NBIA in Switzerland
NBIA scientists will meet to share their research from October 13-15 in Lausanne, Switzerland, at the 8th International NBIA Symposium. More than 20 scientists and physicians from all over the world will present the results of their NBIA projects in...
Research project leads to a stem cell model of FAHN
A team of German scientists who received a $45,000 research grant from the NBIA Disorders Association in 2020 have successfully completed a stem cell model of FAHN to give researchers a better understanding of the disease and...
Pilot Grant in the amount of $69,775 announced for BPAN research
The Million Dollar Bike Ride 2022 (more information on this fundraising campaign) raised $69,775, which the Orphan Disease Center is now offering for a research project on BPAN. Researchers can apply for a pilot project at the following link.
Cycling for BPAN - Million Dollar Bike Ride 2022 a complete success again!
Video: NBIA DA It worked again - in 2022, the Million Dollar Bike Ride (MDBR, https://www.milliondollarbikeride.org/) of the University of Pennsylvania in Philadelphia was able to far exceed the US$ 20,000 mark: Almost 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 at the youth hostel in 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 accompanied by restricted movement, alternating muscle tension and involuntary cramps (dystonia), which cause the muscles to become increasingly shortened. Under certain conditions, myofasciotomy can provide some relief.
CoA Therapeutics press release on Phase 1 study for PKAN
We hereby issue the following press release from CoA Therapeutics: "CoA Therapeutics and BridgeBio Pharma present data from the Phase 1 study in healthy individuals to support the development of BBP-671 for PKAN and organic acidemia On May 26, 26th...
CoA Therapeutics presents timeline towards 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 of healthy...
Survey Helps Prepare Clinical Trial for PKAN Patients
The Californian company CoA Therapeutics is currently preparing a clinical trial of a new drug for PKAN patients. In order 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) and Emily (5 years). 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 an 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 Munich Outpatient Children's Hospice Foundation to mark Rare Disease Day 2022:...
Million Dollar Bike Ride research grant awarded for 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 announced $66,366 will be awarded to Young-Ah Seo of the...
Current call for MPAN funding reflects clearly defined priorities
Hoffnungsbaum e.V. is partnering with three sister organizations on a recent call for funding of up to USD 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...
Generational change in the Management Board of Hoffnungsbaum
On 21.11.2021, the annual general meeting of Hoffnungsbaum e.V. (HoBa) – with groundbreaking decisions for the future of our NBIA patient organization through the completion of the generational change in the association's management and the reorganization...
Guideline with recommendations for the treatment of BPAN available
Researchers from various medical institutions around the world, with the support of affected families, have recently published a guide to the treatment of beta propeller protein-associated neurodegeneration (BPAN). The publication serves as a...
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 study. Scientists led by Dr. Holger Prokisch from the Technical University of Munich have developed a...
PKAN drug screening at Yale University
Since July 2021, a new one-year PKAN research project has been underway at Yale University, New Haven (USA) under the direction of Professor Choukri Ben Mamoun. The project, entitled: "A high-throughput screen for PKAN-revising agents", is being carried out as part of...
RARE-X: A new, secure database for the exchange of BPAN health data
BPAN sufferers and their relatives are invited to post health information about the disease on the new, secure platform RARE-X. The purpose of this database is to collect information about rare diseases such as BPAN and to use it for research into the development of...
Million Dollar Bike Ride again successful with support from Hoffnungsbaum
The Million Dollar Bike Ride 2021 charity bike race at the University of Pennsylvania in Philadelphia, USA, was once again held virtually this year. It serves to raise funds for research into rare diseases, which this year will be supported by 52...
Lena Burbulla discovers new possible BPAN disease mechanisms
Prof. Dr. Lena Burbulla recently successfully completed a project to investigate beta-propeller protein-associated neurodegeneration (BPAN). The project, 2019 with a total budget of €65,000 from Aisnaf, NBIADA and Hoffnungsbaum e.V. together...
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 indicates in the name of the company...
NBIA Disorders Association family conference sessions now available online
The virtual conference platform is now open to all, free of charge, so that interested families with English language skills can watch 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 launched a fundraising campaign in our garden at home in aid of BPAN. Friends, relatives and acquaintances were invited to come by on their bikes and make a donation. In return, we served 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 not only take place 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 to collect donations for the PKAN Fund at Hoffnungsbaum for 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 for our families, especially in German-speaking countries. A loose sequence of video meetings on various topics relevant to NBIA sufferers will take place....
Review of TIRCON and its importance for NBIA research and care
The journal "Frontiers in Neurology" has now published a retrospective article on TIRCON (Treat Iron-Related Childhood-Onset Neurodegeneration) on the topic of "Networks for Movement Disorders". From 2011 to 2015, TIRCON is a project funded by the European Union...
Researcher at Yale University is working on a new therapeutic approach for PKAN
We received news from the USA that a possible new treatment 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 required to...
Hoffnungsbaum Info Letter 2020
Also in 2020 we have put together an information letter for you. It contains information about the association's activities in 2020 and an outlook for next year. If you want to be kept up to date, subscribe to our online newsletter.
PKAN family launches another fundraising campaign
As at the beginning of the year, the Bianchi family is once again actively collecting donations for PKAN research during Advent, as their 3-year-old son Giulio suffers from this form of NBIA. In their home town of Kronau, parents Melanie and Tony have set up a stand with...
Record number of participants at the 7th International Scientific NBIA Symposium
Authors of the original article: Patricia Wood and Angelika Klucken 160 participants from 26 countries attended the 7. International Symposium on NBIA and Related Diseases, which was held as a virtual event for the first time. Since online access makes it easy to...
Interim results of four BPAN research projects published
The NBIA Disorders Association, our sister organization in the USA, reports in its autumn newsletter on the progress made so far 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. conducts a wide-ranging opinion poll among all members of the association 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
The Bianchi family was diagnosed with PKAN last year for their now 3-year-old son Giulio. The parents then launched a fundraising campaign via PayPal and a Christmas card campaign in the private environment for the benefit of PKAN research and are making a Christmas card campaign in the private environment.
Hoffnungsbaum e.V. finances MPAN project at Helmholtz Zentrum München
Hoffnungsbaum e.V. awards funding in the amount of € 151,540 to Dr. Arcangela Iuso and her team from the Institute of Neurogenomics at the Helmholtz Centre in Munich. The funding will finance a 27-month research project aimed at uncovering the essential...
Research team from Rostock receives grant for FAHN-research
Our sister organization in the USA, the NBIA Disorders Association, has awarded a $45,000 grant for research into 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 of the University of Pennsylvania in Philadelphia in the USA, the Million Dollar Bike Ride 2020, took place under difficult conditions. Its purpose is to raise funds for research into rare diseases.
NBIA Symposium 2020 takes place online
The 7. International Symposium on NBIA and Related Diseases will now be held virtually from September 30 to October 3, 2020. Save the date for our virtual symposium! • Update on new findings in NBIA diseases...
7th International NBIA Symposium in Lausanne postponed!
Postponed to autumn 2021 due to the Corona situationThe 7th International NBIA Symposium will unfortunately not take place from 1 – 3 October 2020 in Lausanne, Switzerland and will be postponed to autumn 2021. For three days, scientists and physicians from...
Hoffnungsbaum Information letter at the end of 2019
Hoffnungsbaum e.V. sent out a newsletter as a print edition for the last time this winter, which is available for download here: Hoffnungsbaum-Infoletter-Winter-2019 In the future, we will mainly be able to use our new website or our website.
New research project on BPAN in Tübingen
Tübingen research group led by Professor Tassula Proikas-Cezanne and Hoffnungsbaum e.V. join forces to research BPAN Deciphering the causes of rare, neurodegenerative diseases is a lengthy process that involves a great deal of effort for those...
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 and in my role as Second Chairman of Hoffnungsbaum e.V.I took part in a public panel discussion on the topic of "Autophagy and its importance for health and neurodegeneration". You...
First results from FORT study for PKAN
The pharmaceutical company Retrophin had developed the drug fosmetpantotenate for the treatment of PKAN. This was evaluated for safety and efficacy in a placebo-controlled and multicenter double-blind clinical trial (FORT study).
BPAN research project funded: Iron and neurodegeneration
In 2019, our Italian sister organization AISNAF once again led an international call for proposals 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 of Clinical Trial with Deferiprone
From 2011 to 2015, the international NBIA research project TIRCON (Treat Iron-Related Childhood-Onset Neurodegeneration) received financial support from the European Union. A key TIRCON sub-project was a clinical trial to investigate the safety of...
"Together strong for Maya" fundraising campaign for MPAN research
A large charity event was held in Lauterbach on June 1, 2019. The fate of 12-year-old Maya, who is suffering from the NBIA variant MPAN, moves the whole town, many other people in Saarland and beyond. They all want Maya and...
MPAN can also be inherited in an autosomal dominant manner
The NBIA research group of Dr. Susan Hayflick at Oregon Health & Science University in Portland (OR, USA) has discovered 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 tender
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 for their research on PKAN zebrafish. As part of the 2018 joint international funding initiative, the project led by Lauriel Earley...
Call for proposals for the funding of NBIA research projects
The NBIA patient organizations AISNAF (Italy), Hoffnungsbaum e.V.(Germany) and NBIA Disorders Association (USA) announce a joint funding initiative to award research grants for the study of the following two NBIA variants:...
Half marathon turned into a BPAN charity run
Participants of the MPIA at this year's SAS Half Marathon in Heidelberg, Image: MPIA/J. Henshaw On April 7, 2019, 14 colleagues from our board member Markus Nielbock from the Max Planck Institute for Astronomy (MPIA) made a...
Rhein-Neckar-Zeitung reports on Milly's mission
February 28 is International Rare Disease Day. Numerous events have been taking place around the globe to mark the occasion for days and continue to do so today. There is also a great deal of interest from the media. The Day of Rare Diseases is intended to...
News from Maya and the MPAN fundraiser
In December, we presented the Matthiesen family's fundraising campaign for MPAN research. Maya herself actively supports this crowdfunding campaign, which was launched by her parents shortly after her diagnosis. Together with her big brother, she has...
Rare Disease Day in Heidelberg and Ulm 2019
February 28 is 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, the Organization for Rare Diseases in Germany...
Three NBIA patient organizations support 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
On 29 November, the Saarländischer Rundfunk (SR) drew attention to the family's situation and their fundraising campaign in an empathetic television report as part of its magazine "Wir im Saarland". It was only in September before that the Matthiesen family had been diagnosed...
8th NBIA Family Conference in Germany 2018
From September 21 to 22, 2018, our 8th NBIA Family Conference took place in Düsseldorf with more than 75 participants. In addition to family members and those affected, the participants included doctors, 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 Accumulation (NBIA) and Related Diseases. Around 70 doctors, 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 closing conference on 15 and...
Hertie Foundation's Commitment Award goes to Hoffnungsbaum e.V.
The non-profit Hertie Foundation has once 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...
