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BPAN research project in Cologne - inspired by reports from families
Five NBIA patient organizations from Germany, Italy, the Netherlands, Poland and the USA have joined forces to fund a research project with 100,000 euros over two years. The project is investigating a possible mechanism for a temporary...
PKAN online meeting on 25.09.2024 - register now!
Dear families, the exchange between those affected and their families can be incredibly valuable. Over time, those affected accumulate an enormous amount of knowledge from which others could also benefit. That's why Hoffnungsbaum has launched a new format that...
Demands for resignation take effect - KV boss Heckemann is removed from office
In an open letter, our umbrella organization, the Alliance of Chronic Rare Diseases (ACHSE) e.V., had called for the dismissal of Klaus Heckemann, until then Chairman of the Board of the Association of Statutory Health Insurance Physicians in Saxony, following his controversial...
One gift donation becomes two
For some time now, it has been possible to generate a gift donation using the donation form on our website. You donate as normal, but you can also generate a certificate with various motifs and specify a sender and recipient of the donation.
NBIA gene therapy updates
Historic $1.2 million donation provides fuel for PKAN gene therapy An impressive $1.2 million donation has enabled pantothenate kinase-associated neurodegeneration (PKAN) gene therapy to take a major step forward....
Dr. Ivano Di Meo receives $100,000 grant for INAD research
The NBIA Disorders Association is pleased to announce the results of a $100,000 grant solicitation to support research into PLA2G6-associated neurodegeneration (PLAN). For this funding initiative, the...
Global collaboration leads to progress in MPAN research
In November 2022, Hoffnungsbaum e.V. , together with its sister organizations NBIA Disorders Association (NBIA DA) in the USA, Associazione Italiana Sindromi Neurodegenerative da Accumulo di Ferro (AISNAF) in Italy and Stichting Ijzersterk in the Netherlands, awarded...
CoA Therapeutics discontinues clinical trial for PKAN
It is with a heavy heart that we inform the community that CoA Therapeutics has decided to discontinue the clinical trial of BBP-671, the compound the company had developed as a potential treatment for pantothenate kinase-associated neurodegeneration (PKAN)....
Two BPAN research projects funded by MDBR 2023 with $60,000 each
The 10th annual Million Dollar Bike Ride (MDBR), hosted by the University of Pennsylvania's Orphan Disease Center, resulted in two NBIA researchers each receiving a $60,000 research grant to study the...
Advancing PKAN gene therapy: a collaborative initiative
Research into a gene therapy for pantothenate kinase-associated neurodegeneration (PKAN) continues to progress thanks to the commitment of experienced researchers and newly established partnerships. In 2019, a significant step was taken in PKAN research.
NBIAcure prepares application for FDA approval for CoA-Z
NBIAcure, a research team led by Dr. Susan Hayflick and Dr. Penny Hogarth and based at Oregon Health & Science University (OHSU), has provided the pantothenate kinase-associated neurodegeneration (PKAN) community with an important...
Update on the CoA-Z clinical trial
In recent months, the OHSU CoA-Z study team has shared the initial study results with all participants in the CoA-Z study and their families, as well as with the broader NBIA community of families, friends, clinical providers, and supporters. What follows is...
International scientific NBIA conference in Poland
On October 19 and 20, 2023, a BPAN workshop, an NBIA-Alliance meeting and an international scientific NBIA conference took place in Warsaw, Poland. Researchers, physicians and patient representatives from many countries came together to learn about...
Videos of the NBIA Family Conference 2023 in the USA online
For the first time since the pandemic, the NBIA Disorders Association hosted the 12th NBIA International Family Conference in the United States. For 4 days from May 18 to 21, 2023, a total of 70 NBIA families and 41 speakers, including some NBIA experts, came to Houston, Texas...
Insights into BPAN research at the family conference in the USA
BPAN families who attended the NBIA Disorders Association (NBIADA) family conference in Houston, Texas in May received current insights into BPAN research from four researchers. Kira Anderson, a study coordinator at...
MDBR sets donation record and makes 2 scholarships possible
The NBIA Disorders team and NBIA families raised a record amount for BPAN research this year as part of the University of Pennsylvania's (UPenn) Million Dollar Bike Ride (MDBR). You can find more information about this charity bike race here:...
BPAN researcher develops stem cell model and awaits funding for planned drug screening
Dr. Paul Lockhart from the Murdoch Children's Research Institute in Melbourne, Australia, says that while his BPAN research has developed an important “brain cell” model using stem cells from affected BPAN individuals, his next step is...
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 contents were well received by the Lions Club...
International NBIA Symposium took place in Switzerland in October 2022
Due to the pandemic, the last NBIA scientist meeting in 2020 was held online only. In October 2022, numerous players in NBIA research finally met again in person for the first time since 2017 for 3 days at the 8th International NBIA Symposium in...
Care guidelines for PLAN/INAD are now in progress
Translation of an English article by Patricia Wood Thanks to the support of four organizations that are funding the project, consensus guidelines are now available for the treatment and care of a form of NBIA known as PLAN or PLA2G6-associated...
INAD gene therapy moves one step closer
Shortened translation of an English article by Patricia Wood Work on a gene therapy for Infantile Neuroaxonal Dystrophy, called INAD, received a major boost in October when a London-based biotech company announced its intention to help advance the...
PKAN study with CoA-Z completed in the USA
From 2019 to 2022, Oregon Health & Science University in Portland (USA) conducted an initial clinical trial in the USA for PKAN patients using a new preparation called “CoA-Z” to treat PKAN. This first part of the study has now been...
TV report on 3sat about rare diseases and BPAN
On Rare Disease Day, 28.02.2023, the 3sat science program NANO was dedicated to rare diseases using the example of BPAN. As part of the program, a film crew visited the Nielbock family and their daughter Milly (https://www.millys-mission.de) at home....
Two MPAN project grants totaling $140,000 awarded
Hoffnungsbaum e.V., in partnership with three sister organizations in Europe and the USA, has awarded two MPAN fellowships 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 "Gailingen concept for dealing with swallowing disorders (not only) in children" during a rehabilitation stay of my son in the Hegau-Jugendwerk. Our son benefited greatly from it and so I am pleased that we can welcome 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 neurological development 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 exchange information about their research from October 13-15 in Lausanne, Switzerland as part of the 8th International NBIA Symposium. More than 20 scientists and physicians from all over 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, who received a $45,000 research grant from the NBIA Disorders Association in 2020, has successfully created 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
As part of the Million Dollar Bike Ride 2022 (More information about this fundraising event), $69,775 was raised, which has now been advertised by the Orphan Disease Center for a research project on BPAN. Researchers can apply for a pilot under the following link...
Cycling for BPAN - Million Dollar Bike Ride 2022 a complete success again!
Video: NBIA DA It worked again - in 2022, the Million Dollar Bike Ride (MDBR, https://www.milliondollarbikeride.org/) of the University of Pennsylvania in Philadelphia was able to far exceed the US$ 20,000 mark: Almost US$ 39,000 was raised...
NBIA Family Conference 2022 postponed!
Hoffnungsbaum e.V. wanted to host the 9th NBIA Family Conference in Germany in September 2022 at the Heilbronn Youth Hostel. We were very much looking forward to finally being able to get an in-person meeting of NBIA families again after two years of pandemic.
July 6, 2022, online lecture: Myofasciotomy - a minimally invasive treatment for shortened muscles
NBIA disorders are often associated with movement restrictions, fluctuating muscle tension, and involuntary cramps (dystonia), which increasingly shorten the muscles. Under certain conditions, myofasciotomy can provide some relief here...
Press release from CoA Therapeutics on Phase 1 study for PKAN
We hereby publish the following press release from CoA Therapeutics: "CoA Therapeutics and BridgeBio Pharma Present Phase 1 Healthy Volunteer Data Supporting the Development of BBP-671 for PKAN and Organic Acidemias On May 26...
CoA Therapeutics presents timeline towards PKAN clinical trial
CoA Therapeutics, an affiliate of BridgeBio, has completed a Phase I study with healthy volunteers examining the safety, tolerability, and drug-like properties of BBP-671. We present the data from healthy...
Survey helps prepare clinical trial for PKAN patients
The Californian company CoA Therapeutics is currently preparing a clinical trial with a new drug for PKAN patients. To make this study as patient-oriented 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
In an interview, Noah Rusch shares insights into the challenges and family life with his son Laurin, who suffers from BPAN. The interview was published by the Ambulantes Kinderhospiz München Foundation for 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 advertised $66,366 will be awarded to Young-Ah Seo from the...
Current call for MPAN funding reflects clearly defined priorities
Hoffnungsbaum e.V. is cooperating with three sister organizations on a current call for funding of up to USD 145,000 for a 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 on the Board of Hoffnungsbaum
On November 21, 2021, the annual general 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 management and the reorganization...
Guideline with recommendations for the treatment of BPAN available
Researchers from various medical institutions worldwide, with the support of affected families, have 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 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 running at Yale University, New Haven (USA) under the direction of Professor Choukri Ben Mamoun. The project, entitled: “A High-Throughput Screening for PKAN-Reversing Agents” is being conducted as part of...
RARE-X: A new, secure database for the exchange of BPAN health data
BPAN patients and their relatives are invited to enter health information about the disease on the new, secure RARE-X platform. This database is used to collect information about rare diseases such as BPAN and make it available to research for...
Million Dollar Bike Ride again successful with the support of Hoffnungsbaum
Also this year, the charity bike race of the University of Pennsylvania in Philadelphia in the USA, the Million Dollar Bike Ride 2021, was held virtually. It is used to collect donations for research into rare diseases, which this year will be funded by 52...
Lena Burbulla discovers new possible BPAN disease mechanisms
Prof. Dr. Lena Burbulla was recently able to successfully complete a project to research Beta-Propeller Protein Associated Neurodegeneration (BPAN). The project, with a total budget of €65,000 in 2019, was jointly...
News from CoA-Therapeutics: Phase 1 trial with new drug for PKAN has started
There is current news from CoA Therapeutics, Inc., a subsidiary of BridgeBio Pharma, Inc. The Californian company is working on a new potential therapy for PKAN patients. “CoA” stands for Coenzyme A and indicates in the company name...
NBIA Disorders Association family conference sessions now available online
The virtual conference platform is now open to everyone free of charge, so interested families with knowledge of English can watch those sessions that are available as recordings. The program includes patient-centered 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 for BPAN. Friends, relatives and acquaintances were invited to come by by bike and bring a donation. In return, there was fresh...
Cycling for BPAN - Million Dollar Bike Ride 2021
It's that time of year 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 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 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. It is intended to be a loose series of video meetings on various topics relevant to those affected by NBIA....
Review of TIRCON and its significance for NBIA research and care
Regarding the topic of "Networks for Movement Disorders", a retrospective article about TIRCON (Treat Iron-Related Childhood-Onset Neurodegeneration) has now been published in the journal "Frontiers in Neurology". TIRCON was funded by the European Union from 2011 to 2015...
Researcher at Yale University works on new therapeutic approach for PKAN
We received news from the USA that a possible new therapy option for PKAN is being researched at Yale University. This new approach is still in the very early stages of development, and a great deal of scientific work and time is required to...
Hoffnungsbaum newsletter 2020
We have compiled another information letter for you in 2020. It contains information about the association's activities in 2020 and an outlook for the coming 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 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 English article: Patricia Wood and Angelika Klucken 160 participants from 26 countries attended the 7th International Symposium on NBIA and related disorders, which took place as a virtual event for the first time. Since the online access...
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 to date in four research projects on Beta-Propeller Protein-Associated Neurodegeneration (BPAN). BPAN, one of the...
Results of the 2020 Hoffnungsbaum
Between August and September 2020, Hoffnungsbaum e.V. conducted a broad opinion survey among all 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 fundraising campaign generates more than €15,000 for PKAN research
Last year, the Bianchi family received a diagnosis of PKAN for their son Giulio, who is now 3 years old. As a result, the parents launched a fundraising campaign via PayPal and a Christmas card campaign in their private lives for the benefit of PKAN research and are...
Hoffnungsbaum e.V. finances MPAN project at the Helmholtz Center Munich
Hoffnungsbaum e.V. is awarding funding of €151,540 to Dr. Arcangela Iuso and her team from the Institute of Neurogenomics at the Helmholtz Center in Munich. The funding will finance a 27-month research project to uncover essential...
Research team from Rostock receives grant for FAHN
Our sister organization in the USA, the NBIA Disorders Association, has awarded a $45,000 grant to research 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 University of Pennsylvania charity bike race in Philadelphia in the USA, the Million Dollar Bike Ride 2020, took place under difficult conditions. It is used to collect donations for the research of rare...
NBIA Symposium 2020 takes place online
The 7th International Symposium for NBIA and related disorders will now take place virtually from September 30 to October 3, 2020. Save the date for our virtual symposium! • News on new findings in NBIA diseases...
7th International NBIA Symposium in Lausanne postponed!
Postponed to autumn 2021 due to the corona situation. The 7th International NBIA Symposium will unfortunately not take place from October 1 - 3, 2020 in Lausanne, Switzerland and will be postponed to autumn 2021. For three days, scientists and doctors from...
Hoffnungsbaum newsletter 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-Infobrief-Winter-2019 In the future, we will primarily communicate via our new website or via our...
New research project on BPAN in Tübingen
The research group from Tübingen led by Professor Tassula Proikas-Cezanne and Hoffnungsbaum e.V. are joining forces to research BPAN. Deciphering the causes of rare, neurodegenerative diseases is a lengthy process that involves a great deal for those affected...
PKAN clinical trial in the USA and Canada begins in December 2019
For several years, the research teams of Dr. Susan Hayflick (Oregon University, 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 a Hoffnungsbaum 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". She...
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 clinical double-blind 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 completed at the end of July with the financing of a new research project on BPAN. Thanks to the joint...
PKAN: Results of 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 sub-project was a clinical study to investigate the safety...
"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 being supported by AISNAF, Hoffnungsbaum e.V. and NBIA Disorders Association for their research on PKAN zebrafish. As part of the joint international funding initiative in 2018, 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) are announcing a joint funding initiative for the awarding of research grants for the investigation of the following two NBIA variants:...
Half marathon becomes 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 of our board member Markus Nielbock from the Max Planck Institute for Astronomy (MPIA) took part in the...
Rhein-Neckar-Zeitung reports on Milly's mission
February 28 is International Rare Disease Day. Numerous events have been taking place around the world for days now to mark the occasion. Media interest is also high. The aim of Rare Disease Day is to...
News from Maya and the MPAN fundraising campaign
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 older brother, she...
Rare Disease Day in Heidelberg and Ulm 2019
February 28th is Rare Disease Day, which is celebrated around the world. In cooperation with the Alliance of Chronic Rare Diseases – ACHSE e.V., the umbrella organization for rare diseases in Germany, events are taking place for this occasion...
Three NBIA patient organizations support PKAN gene therapy project
The first international call for research projects from 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 gene therapy for PKAN...
Matthiesen family launches fundraising campaign for MPAN research
Saarländischer Rundfunk (SR) drew attention to the family's situation and their fundraising campaign in a sensitive television report on November 29 as part of its "Wir im Saarland" magazine program. It was only in September that the Matthiesen family had been diagnosed with...
8th NBIA Family Conference in Germany 2018
From 21 to 22 September 2018, our 8th NBIA Family Conference took place in Düsseldorf with more than 75 participants. In addition to family members and those affected, these included doctors, scientists and a team of youth workers. Most of the guests...
News from Milly and her mission
On 22.12.2017, 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, her parents...
Progress at NBIA Symposium & NBIA Alliance Meeting
The 6th International Symposium on Neurodegeneration with Brain Iron Accumulation (NBIA) and related diseases presented the latest findings from research. 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 large final conference on the 15th and...
Hertie Foundation Engagement Award goes to Hoffnungsbaum e.V.
The non-profit Hertie Foundation has once again awarded the prize for commitment and self-help. The prize recognizes model activities of self-help groups and particularly committed people in the field of multiple sclerosis and neurodegenerative...
