The PKAN physician-scientists at OHSU and scientists from the Horae Gene Therapy Center at UMass Chan Medical school have recently formed a partnership to develop gene therapy for PKAN. Early work in PKAN mice was initially done by Dr. Lauriel Earley, a former graduate student in Molecular & Medical Genetics at OHSU. Dr. Earley’s work […]

With the new year, we’d like to send an update on our progress with CoA-Z. In September, we told you that we had decided to set aside plans for further clinical trials with CoA-Z, instead focusing our efforts on compiling the necessary data to present to the FDA in order to gain approval to make […]

At the NBIA Family Meeting in May and in our follow-up communication to CoA-Z study participants, we mentioned the possibility of our doing one or more short follow-up studies of CoA-Z to supplement our clinical trial data. After further data analysis and reflection, we have decided that our current data is strong enough to submit […]

It’s hard to believe it, but the CoA-Z clinical trial started way back in December 2019 when we had no idea how much all our lives were about to be upended by a global pandemic. Many of you have reached out since then to ask for updates and to find out when results will be […]

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 has also written a FAQs […]

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 families. The paper is now […]

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 […]