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 […]

A recent scientific paper (linked below) reports that PLAN is associated with ceramide accumulation in a fruit fly model of this disorder. Ceramide is a type of fat (lipid) used widely in the body, especially in the membranes of cells. The authors report that accumulation of ceramide in the nervous system puts stress on an […]

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 […]

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 […]