This article and other news can be found in the latest issue of our newsletter which can be downloaded/viewed here: April 2018. Back in April 2015, the very first beta-testers started participating in our online natural history study, PKANready. These early testers helped us work out the bugs and finalize the design before the full […]

Our team will now be publishing a newsletter several times a year to update you on our research studies and let you know about upcoming projects. If you would like to join the mailing list to receive the pdf by email as soon as it’s released, please let us know by sending an email to […]

As promised, here are updates on the fundraising questions you had about CoA-Z, the possible treatment of PKAN that we are developing. 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 […]

Is it true that OHSU is working on a treatment for PKAN? Many of you in the family community have heard rumors that the OHSU team is working on a new treatment for PKAN, and we know you are eager to learn more. We’ve been keeping very quiet about our work because we are caring […]

Our team is excited to announce that we and several of our collaborators have developed a set of guidelines called “Best Practices in PKAN.” These guidelines provide recommendations and instructions for taking care of a child or adult with PKAN. They will be a valuable resource for patients themselves and for any doctors they may […]

Last year at the family conference, Dr. Hayflick announced that work had begun on a set of guidelines called “Best Practices in PKAN” that would provide recommendations and instructions for taking care of a child or adult with PKAN. This project was funded by the NBIA Disorders Association, Hoffnungsbaum, e.V., and Associazione Italiana Sindromi Neurodegenerative […]

You may recall that back in September we asked if anyone had questions about the PKAN knock-in mice for our resident mouse wrangler, Jeff. We had one curious dad who submitted some very interesting questions (and a few amusing ones that gave our team a good laugh). So without further ado, here are some answers: […]

This has been an exciting month for our mice! In September, a new litter who we have been eagerly anticipating was born in the Hayflick lab. As you may recall from one of the past NBIA Disorders Association newsletters, the Hayflick lab is developing a new mouse model for NBIA. This mouse has been genetically […]

Those of you who attended this year’s NBIA Disorders family conference may recall Dr. Hayflick, Dr. Hogarth and Allison emphasizing how important it is to collect natural history data in order to get the NBIA community ready for any drug trials that may be coming down the pipeline. We are now taking the first step […]

Deferiprone is a medication that chelates, or binds, iron and removes excess amounts from the body. Unlike previous chelator drugs, deferiprone can enter the brain, remove iron from places where it is high, and “redistribute” it. It has been used for treatment of various diseases since 1994 in Europe and Asia. Deferiprone was approved by […]