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 original, subject to charge)
- Website of the NBIA Disorders Association (English)
- GeneReviews® review article on NBIA (with linked articles on the 10 most important NBIA forms, English)
Updates on PKAN
Here you will find the latest information on the NBIA variant PKAN.
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...
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....
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)....
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...
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...
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...
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...
