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 […]

A new medication for Rett syndrome, called Daybue, was recently approved by the FDA. Since there are some clinical similarities between BPAN and Rett syndrome, including regression, seizures, intellectual disability, motor changes and certain repetitive behaviors, it is natural to wonder whether Daybue could help people with BPAN. We at OHSU continue to ask ourselves […]

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 […]

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: August 2018. In 2014 the Hayflick lab started a project with the University of Washington to sequence DNA for our patients who did not yet have a genetic diagnosis. These 54 subjects had symptoms similar […]

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 […]

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 […]