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 […]

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 […]

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 […]

A summary of the 2016 BPAN Research Meeting in Portland is now up on our website! Check it out to learn about what we did, see lots of pictures of the wonderful children and families who attended and read the research updates we shared at the symposium. We can’t say thank you enough to everyone […]

Our team is excited to announce that we will be hosting a BPAN research meeting in Portland, Oregon from June 24th-26th! We are thrilled to welcome you to our hometown and have planned a variety of activities including research activities, social events, a mini-symposium, and individual appointments with Dr. Hayflick and Dr. Hogarth. For more […]

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 […]