PKAN - Pantothenate kinase-associated neurodegeneration.
An updated disease description is currently in progress. Please be patient a little longer.
The most up-to-date patient-focused information on NBIA and its various 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 original)
- NBIA Disorders Association website (English)
- GeneReviews® review article on NBIA (with linked articles on the main 10 NBIA forms, English).
News about PKAN
Here you will find current information on the NBIA variant PKAN.
Fundraising-Aktion des Lions-Club Mühlacker für Hoffnungsbaum e.V.
Mühlacker, Dezember 2022: Im Rahmen eines Club-Abends des Lions-Club Mühlacker hatte das Hoffnungsbaum e.V. Vorstandsmitglied Tiemo Durm die Möglichkeit einen Vortrag über den Verein sowie dessen Ziele und Hintergründe zu halten. Die Inhalte stießen beim Lions-Club...
PKAN study with CoA-Z completed in the USA
Die Oregon Health & Science University in Portland (USA) hat von 2019 bis 2022 mit einem neuen Präparat namens „CoA-Z“ zur Behandlung von PKAN eine erste klinische Studie für PKAN-Patienten in den USA durchgeführt. Dieser erste Studienteil wurde inzwischen...
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 Phase 1 study data in healthy subjects to support the development of BBP-671 for PKAN and organic acidemias On May 26,...
CoA Therapeutics unveils timeline on path to 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 from healthy...
Survey helps prepare clinical trial for PKAN patients
California-based CoA Therapeutics is currently preparing a clinical trial of a new drug for PKAN patients. 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.
A new one-year PKAN research project has been underway at Yale University, New Haven (USA) since July 2021 under the leadership of Professor Choukri Ben Mamoun. The project, entitled: "A High-Throughput Screening for PKAN-Revising Agents," will be conducted in the...
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 in the company's name indicates...
NBIA Disorders Association family conference sessions now available online
The virtual conference platform is now open to all, tuition-free, so interested English-speaking families can view 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 and began fundraising for the PKAN Fund at Hoffnungsbaum to benefit research into one of the most common forms of NBIA, because nothing...
Review of TIRCON and its relevance to NBIA research and care.
On the topic of movement disorder networks, a retrospective article on TIRCON (Treat Iron-Related Childhood-Onset Neurodegeneration) has now appeared in the journal Frontiers in Neurology. From 2011 to 2015, TIRCON is a European Union-funded...
