Discovery clears way for clinical trial to treat devastating neurologic disorder

New compound, tested in mice and human cells, corrects abnormalities that cause the disease

By Erik Robinson October 29, 2019 Portland, Oregon

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Carter Anderson of Butte Falls, holds hands with his father Blair during an appointment at OHSU, late 2017. Carter was diagnosed with PKAN - Pantothenate Kinase-Associated Neurodegeneration, a genetic disease which affects his ability to walk, talk, eat and control basic movements. (OHSU/Kristyna Wentz-Graff)

OHSU/Kristyna Wentz-Graff

Susan Hayflick, M.D., awarded (4)

Carter Anderson of Butte Falls, with his mother Courtney (center) during an appointment at OHSU with Dr. Susan Hayflick in 2017. Carter was diagnosed with PKAN - Pantothenate Kinase-Associated Neurodegeneration, a genetic disease which affects his ability to walk, talk, eat and control basic movements. (OHSU/Kristyna Wentz-Graff)

OHSU/Kristyna Wentz-Graff

PKAN

Carter Anderson of Butte Falls was diagnosed with PKAN - Pantothenate Kinase-Associated Neurodegeneration, a genetic disease which affects his ability to walk, talk, eat and control basic movements. (OHSU/Kristyna Wentz-Graff)

OHSU/Kristyna Wentz-Graff

PKAN

(Left to right) Carter Anderson of Butte Falls, with his parents Courtney and Blair Anderson, during an appointment at OHSU in late 2017. Carter was diagnosed with PKAN - Pantothenate Kinase-Associated Neurodegeneration, a genetic disease which affects his ability to walk, talk, eat and control basic movements. (OHSU/Kristyna Wentz-Graff)

OHSU/Kristyna Wentz-Graff

PKAN

Carter Anderson of Butte Falls, with his mother Courtney during an appointment at OHSU in late 2017. Carter was diagnosed with PKAN - Pantothenate Kinase-Associated Neurodegeneration, a genetic disease which affects his ability to walk, talk, eat and control basic movements. (OHSU/Kristyna Wentz-Graff)

OHSU/Kristyna Wentz-Graff

PKAN

Carter Anderson of Butte Falls, with his mother Courtney, during an appointment at OHSU, late 2017. Carter was diagnosed with PKAN - Pantothenate Kinase-Associated Neurodegeneration, a genetic disease which affects his ability to walk, talk, eat and control basic movements. (OHSU/Kristyna Wentz-Graff)