Zbigniew K. Wszolek, M.D.

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. 

The purpose of this study is to evaluate the safety and tolerability of VGL101 for the treatment of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and potential effectiveness of multiple doses of ONO-2808 in patients with MSA.  

The purpose of this study is to assess the impact of AMX0035 compared to placebo on disease progression rate as measured by the Progressive Supranuclear Palsy (PSP) Rating Scale (PSPRS).

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.

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.

The purpose of this study is to measure the effect of Hematopoietic Stem Cell Transplantation (HSCT) on symptoms of CSF1R-related Leukoencephalopathy.

The purpose of this study is to evaluate the dose-related safety of BIIB054, to evaluate the pharmacodynamic effects of BIIB054 on the integrity of nigrostriatal dopaminergic nerve terminals, to assess the pharmacokinetic (PK) profile of BIIB054 and to evaluate the immunogenicity of BIIB054.

The purpose of this study is to compare the effectiveness of BHV-4157 (200mg once daily) versus placebo after 48 weeks of treatment in subjects with spinocerebellar ataxia (SCA).

The purpose of this study is to assess the effectiveness, safety, tolerability, and drug/body interactions of ABBV-8E12 for the treatment of patients who have progressive supranuclear palsy.

The purpose of this study is to compare the effectiveness of BHV-3241 versus placebo in subjects with Multiple System Atrophy.

The purpose of this study is to assess the long-term safety and effectiveness of ABBV-8E12 in subjects with progressive supranuclear palsy (PSP).

To learn more about the biomarkers for preclinical parkinson's disease. 

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.

This study is being done to collect skin samples from people with and without neurodegenerative and vascular disorders including Parkinson’s disease (PD), Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS), stroke and many others. We will use these skin samples to make and bank (store) a group of cells (cell line) called inducible pluripotent stem (iPS) cells.

The purpose of this study is to compare the sensitivity and specificity of ultra-high field MRI (7 Tesla) to standard 3T MRI for the of characteristic imaging findings in the diagnosis of Adult-onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP).