Open-label add-on treatment trial of minocycline in fragile X syndrome |
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| Authors: | Carlo Paribello Leeping Tao Anthony Folino Elizabeth Berry-Kravis Michael Tranfaglia Iryna M Ethell Douglas W Ethell |
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| Affiliation: | 1.Surrey Place Centre,Toronto,Canada;2.Departments of Pediatrics, Neurological Sciences, Biochemistry,Rush University Medical Center,Chicago,USA;3.FRAXA Research Foundation,Newburyport,USA;4.Biomedical Sciences,University of California,Riverside,USA;5.Biomedical Sciences,Western University of Health Sciences,Pomona,USA |
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| Abstract: | Background Fragile X syndrome (FXS) is a disorder characterized by a variety of disabilities, including cognitive deficits, attention-deficit/hyperactivity disorder, autism, and other socio-emotional problems. It is hypothesized that the absence of the fragile X mental retardation protein (FMRP) leads to higher levels of matrix metallo-proteinase-9 activity (MMP-9) in the brain. Minocycline inhibits MMP-9 activity, and alleviates behavioural and synapse abnormalities in fmr1 knockout mice, an established model for FXS. This open-label add-on pilot trial was conducted to evaluate safety and efficacy of minocycline in treating behavioural abnormalities that occur in humans with FXS. |
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