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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 has developed a possible therapeutic for PKAN called CoA-Z through collaboration with Dr. Sibon in the Netherland. 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 move the compound to clinical trial. The link above contains information about CoA-Z and how you can send donations to the foundation.

coaztriallogo-2 CoA-Z Clinical Trial
The US/Canada clinical trial for CoA-Z is now enrolling! This page gives an overview of the trial and how you can get in touch with our team to find out more and/or enroll. For families outside of the US and Canada, we will provide updates on trials in other countries as progress is made and information becomes available.




NBIAcure News

BPAN Management Guideline and Podcast
A consensus management guideline for BPAN has been published in Developmental Medicine & Child Neurology that will help support clinicians by guiding the diagnostic process, management and surveillance at different ages and stages of BPAN. We hope it will serve as a valuable resource for families who often become advocates...
Update on BPAN
A scientific paper about BPAN recently published in the journal Autophagy has created some confusion within the community of BPAN families, friends, and caregivers. Entitled “WDR45, one gene associated with multiple developmental disorders,” the paper suggests that gene mutations in WDR45 lead to six separate disorders rather than just BPAN....

  


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