News from research
Here you will find current information from NBIA research and related research areas.
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...
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...
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...
