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Clinical Studies


  • A Natural History Study of Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP) Jacksonville, Fla.

    The purpose of this study is to collect data to contribute to the development of future novel therapies, including VGL101, that focus on the neuropathophysiological features that underlie adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) and that are essential to reverse, delay, or stop progression of this debilitating disorder.

    Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) is a rare, rapidly progressing, genetic, neurodegenerative disease for which no definitive treatment options and limited information on the natural history of the disease are available. The structural, genetic, and neuropathophysiological abnormalities of ALSP lead to the onset of neurologic symptoms, such as moderate to severe motor and neuropsychiatric impairments. 

  • Investigating the Biomarkers for Better Characterization of Phenotypes of Neurodegenerative Disorders Jacksonville, Fla.

    The primary purpose of this study is to create a Mayo Clinic repository for  neurodegenerative disorders such as Parkinson’s disease (PD), Parkinson’s disease dementia and dementia with Lewy bodies (PDD/DLB), multiple system atrophy (MSA), progressive supranuclear palsy (PSP), cortico-basal degeneration (CBD), and other even rarer forms of neurodegeneration.

  • Investigating the Genetic and Phenotypic Presentation of Ataxia and Nucleotide Repeat Diseases Jacksonville, Fla.

    The purpose of this study is to create a repository for cerebellar ataxia and nucleotide repeat diseases in order to fully investigate the genetic and phenotypic presentations of both.

  • Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of 36 Weeks of Treatment With NLY01 in Early-stage Parkinson's Disease Jacksonville, Fla.

    The purpose of this study is assess the safety, tolerability and effectiveness of NLY01 in subjects with early untreated Parkinson's disease (PD). Evidence suggests NLY01, a pegylated form of exenatide, may be beneficial in PD and is being developed as a potential treatment for neurodegenerative disorders.

  • Quantitative Phenotypes of Movement Disorders Jacksonville, Fla.

    The purpose of this study is to determine if adaptive rhythmic auditory stimulation (ARAS) is a safe means of improving gait; i.e., fewer falls, abrupt accelerations/decelerations in patients with movement disorders. Furthermore, we aim to provide patients with a safe environment in which they can safely navigate through an enjoyable experience (guided art tour) that provides exercise, social engagement, and exposure to art.

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