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Welcome to NBIAcure

Although it may be out of reach today, a cure for each of the NBIA disorders is in our sights. We care deeply about improving the lives of children and adults with NBIA. Our team at NBIAcure will provide support and information to patients, families, clinicians and researchers, initiate new research studies to lay the foundation for clinical trials, and collaborate with academic and industry partners to develop new treatments.

We invite you to explore our site and return often to check on the latest news stories, find out about new studies that have launched, and learn how you can get involved. Your participation is one of the keys to the cure.

For the first time ever, people affected by an NBIA disorder can tell us about their disease and how it affects their lives. Better information about how each of these diseases progresses prepares us for therapeutic drug trials. We are building knowledge to determine if new drugs improve health and function. People with an NBIA disorder as well as family members will be able to contribute. Stay connected here and help our community become 'trial ready.'

image1-1 Spoonbill Foundation
Our team is currently developing a possible therapeutic for PKAN called CoA-Z. This compound was created through collaboration with Dr. Sibon in the Netherlands and we believe it may change the course of this terrible disease. Our goal is to make CoA-Z available to everyone at the lowest cost possible so we have formed sister non-profit foundations in the Netherlands and US to further develop the compound. The link above contains information about CoA-Z, the steps pending to get it to clinical trial, the cost associated with each step, and how you can send donations to the foundation.

coaztriallogo-2 CoA-Z Clinical Trial
We will share updates about the US and Canada clinicial trial on this page as we get closer to launch. For families outside of the US and Canada, we will be providing updates on trials in other countries as progress is made and information becomes available.

NBIAcure News

A paper from the Sibon lab reveals important insights into biochemical changes in PKAN and related disorders
Dr. Sibon’s group in the Netherlands has published a paper that reveals important insights into the biochemical changes in PKAN and related disorders (CoPAN, MePAN and PDH-E2 deficiency). It reports work done largely by Roald Lambrechts (who just got his PhD yesterday!) and others in the Sibon lab. Dr. Sibon...
A new paper explains the rationale behind the CoA-Z clinical trial
This peer-reviewed paper authored by our OHSU team in partnership with our Dutch colleagues helps explain the rationale behind our proposed CoA-Z trial in humans. In addition to a link to the paper below, we also developed some Frequently Asked Questions that summarize key points and their relevance for PKAN...


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