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  • A Phase II Study of Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis Rochester, Minn., Jacksonville, Fla., Scottsdale/Phoenix, Ariz.

    The purpose of this study is to determine the safety and efficacy of intrathecal treatment delivered to the cerebrospinal fluid (CSF) of mesenchymal stem cells in ALS patients every 3 months for a total of 4 injections over 12 months. Mesenchymal stem cells (MSCs) are a type of stem cell that can be grown into a number of different kinds of cells. In this study, MSCs will be taken from the subject's body fat and grown. CSF is the fluid surrounding the spine. The use of mesenchymal stem cells is considered investigational, which means it has not been approved by the Food and Drug Administration (FDA) for routine clinical use. However, the FDA has allowed the use of mesenchymal stem cells in this research study.

  • Blood and Spinal Fluid Collection for Research Studies in Motor Neuron Disease and Motor Neuropathy Rochester, Minn.

    The purpose of this study is so that blood (plasma and blood cells) and/or extra spinal fluid (which was taken for clinical purposes) can be obtained and stored for later research on a large cohort of patients with progressive motor neuronopathies and neuropathies, many of which will be determined to suffer from Amyotrophic Lateral Sclerosis (ALS). 

  • Collection of Discarded Cerebrospinal Fluid for Research Purposes Rochester, Minn.

    The purpose of this study is to collect and store cerebrospinal fluid (CSF)from patients with normal CSF.  This stored CSF will be used in the future as a culture media to better understand how therapeutic cells delivered into the CSF will behave. 

  • Immune Phenotyping in Amyotrophic Lateral Sclerosis Rochester, Minn.

    The goals of this study are to identify biomarkers that allow improved staging or prognosis of the disease, and through the discovery of previously unrecognized immune abnormalities in ALS we aim to eventually identify immune therapies that may provide benefit in ALS.

  • Induced Pluripotent Stem Cell Development from Skin Fibroblasts in Patients with Hereditary Peripheral Neuropathy Rochester, Minn.

    This research is being done to develop stem cells from cells within patients’ skin. These stem cells from patients will be used to better understand peripheral neuropathy and help develop treatments.

  • Patient Specific Induced Pluripotent Stem Cell Models for Spinal Muscular Atrophy (SMA) Rochester, Minn., La Crosse, Wis.

    The purpose of this study is to perform RNA sequencing on induced pluripotent stem cells (iPSC).  By comparing the gene expression of motor neurons derived from iPSC cells of phenotypically discordant Spinal Muscular Atrophy (SMA) sibling pairs and individual cases of SMA, we hope to identify potential protective gene modifiers of SMA.   Identifying protective gene modifiers will increase our understanding of the molecular pathogenesis of SMA.

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  • Skin-Neuronal Interactions in Peripheral Neuropathy Rochester, Minn.

    The purpose of this study is to collect skin biopsies from patients who have known cases of chemotherapy induced peripheral neuropathy and analyze the samples microscopically for skin and nerve cell interactions.

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