Nathan P. Staff, M.D., Ph.D.

This is a Phase 1/2 open-label, multicenter study to evaluate the safety and tolerability and to explore the efficacy of gene therapy AMT-162 in Participants with ALS with pathogenic or likely pathogenic variants in the SOD1 gene (SOD1-ALS). Approximately 12-20 Participants with SOD1-ALS will be treated in this study.

The purpose of this study is to provide access to the investigational product, pridopidine, for up to 200 participants with ALS who are ineligible for clinical trials.

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). 

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. 

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.

The purpose of this research is to develop stem cells from cells within patients’ skin in order to better understand peripheral neuropathy and help develop treatments.

The purpose of this study is to establish a repository consisting of clinical information, radiological data, serum, peripheral blood lymphocytes, plasma, cerebrospinal fluid (CSF), urine, stool, DNA and tissue specimens collected from patients with neurological diseases or patients with non-neurological conditions to be used as controls or for future genetic/familial studies.

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.

The purpose of this study is to test the clinical effectiveness of ibudilast in patients with ALS measured by NfL and change in ALSFRS-R slope.

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.

The purpose of this study is to develop and validate a novel diagnostic test for SFN utilizing RNAseq from skin biopsies of the distal leg.

The purpose of this study is to evaluate and compare the effectiveness of two dosing regimens of oral edaravone in subjects with amyotrophic lateral sclerosis (ALS), based on the time from the randomization date in Study MT-1186-A02 to at least a 12-point decrease in Revised ALS Functional Rating Score (ALSFRS-R) or death, whichever happens first, over the course of the study or until oral edaravone is commercially available in that country.

This study aims to evaluate and compare the effectiveness of two dosing regimens of oral edaravone in subjects with amyotrophic lateral sclerosis (ALS) based on the change in ALS Functional Rating Scale. 

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.

The purpose of this study is to assess safety, tolerability, and pharmacokinetics (PK) following multiple doses of ABBV-CLS-7262 in subjects with amyotrophic lateral sclerosis (ALS).

The purpose of this study is to confirm that levosimendan can significantly improve respiratory function measured by supine slow vital capacity (SVC) in amyotrophic lateral sclerosis (ALS) patients.

The purpose of this study is to collect clinical and biomarker data from patients receiving neurotoxic chemotherapy who are at risk for developing Chemotherapy-induced Peripheral Neuropathy (CIPN).

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.