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 will find the latest information on the NBIA variant PKAN.
CoA Therapeutics bricht Klinische Studie für PKAN ab
Übersetzung eines englischen Artikels von Amber Denton, Präsidentin der NBIA Disorders Association, aus dem Newsletter der NBIA Disorders Associacion Schweren Herzens teilen wir der Gemeinschaft mit, dass CoA Therapeutics beschlossen hat, die klinische Studie zu...
Update on the CoA-Z clinical trial
Over the past several months, the OHSU CoA-Z study team has shared the initial study results with all CoA-Z study participants and their families, as well as the broader NBIA community of families, friends, clinical providers and supporters. It follows...
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
Oregon Health & Science University in Portland (USA) conducted the first clinical trial for PKAN patients in the USA from 2019 to 2022 with a new drug called "CoA-Z" for the treatment of PKAN. This first part of the trial has now been...
Press release from CoA Therapeutics on Phase 1 study for PKAN
We hereby issue the following press release from CoA Therapeutics: "CoA Therapeutics and BridgeBio Pharma Present Data from Phase 1 Study in Healthy Subjects to Support Development of BBP-671 for PKAN and Organic Acidemias On May 26,...
CoA Therapeutics presents timeline towards PKAN clinical trial
CoA Therapeutics, a BridgeBio affiliated company, has completed a Phase I study in healthy volunteers evaluating the safety, tolerability and drug-like properties of BBP-671. We present the data from healthy volunteers...
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. In order to make this study as patient-oriented 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
The latest news from CoA Therapeutics, Inc., a subsidiary of BridgeBio Pharma, Inc. is that the Californian company is working on a new potential therapy for PKAN patients. "CoA" stands for Coenzyme A and the company's name already...
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...
