PKAN – Pantothenate kinase-associated neurodegeneration
An updated description of the disease is currently being prepared. Please be patient.
The latest patient-oriented information on NBIA and the individual forms can be found here:
- Patient-oriented disease description from the ACHSE network (German)
- PKAN Consensus Recommendations (German)
- PKAN Consensus Recommendations (English manuscript of the paid originals)
- Website of the NBIA Disorders Association (English)
- GeneReviews® review article on NBIA (with linked articles on the 10 most important NBIA forms, English)
News about PKAN
Here you can find the latest information on the NBIA variant PKAN.
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...
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...
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...
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...
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...
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...
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...
