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Susan Hayflick, MD
OHSU
Medical geneticist whose team identified the gene responsible for PKAN and has led decades of PKAN research.
research + treatment
What is happening now
PKAN research is moving forward. This page is a simple place to understand the work underway, who is doing it, and where families can learn more.
ACTIVE RESEARCH
Gene therapy
A major PKAN gene therapy program is being advanced through a collaboration between physician-scientists at Oregon Health & Science University and researchers at the Horae Gene Therapy Center at UMass Chan Medical School.
The goal is to develop an AAV-based therapy that delivers a working copy of the PANK2 gene to the central nervous system. The teams bring together decades of PKAN knowledge, gene-delivery expertise, animal-model research, clinical-trial experience, and FDA translation.
fundraising goal
$5 million
$2 million raised by Loving Loic as reported in 2026
people behind the work
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Penny Hogarth, MD
OHSU
Movement-disorders neurologist and clinical-trial investigator with extensive experience caring for people with PKAN.
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Miguel Sena-Esteves, PhD
UMass Chan
Gene-therapy scientist specializing in AAV-based treatments and delivery to the central nervous system.
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Heather Gray-Edwards, DVM, PhD
UMass Chan
Researcher developing and testing gene therapies, including new AAV vectors and delivery approaches.
OTHER APPROACHES
Beyond gene therapy
Gene therapy is not the only path being explored. Researchers have also investigated ways to address the downstream effects of PANK2 dysfunction and restore the cellular pathways affected in PKAN.
As new studies, trials, and treatment programs emerge, we can add them here so families have one clear place to see what is happening.
how to use this page
Follow the work, not the noise.
Research changes. We’ll date updates and link directly to the sources.
last reviewed · august 2026