A5087544782- Amyotrophic Lateral Sclerosis Research
- Neurogenetic and Muscular Disorders Research
- Parkinson's Disease Mechanisms and Treatments
- Prion Diseases and Protein Misfolding
- Neurological diseases and metabolism
- Alzheimer's disease research and treatments
- Pesticide Exposure and Toxicity
- Genetic Neurodegenerative Diseases
- Cholinesterase and Neurodegenerative Diseases
- Biochemical Acid Research Studies
- Cancer-related gene regulation
- Spinal Cord Injury Research
- Hereditary Neurological Disorders
- Neuroinflammation and Neurodegeneration Mechanisms
- Statistical Methods and Inference
- Dysphagia Assessment and Management
- Palliative Care and End-of-Life Issues
- Pluripotent Stem Cells Research
- biodegradable polymer synthesis and properties
- Neurological and metabolic disorders
- Statistical Methods and Bayesian Inference
- Cystic Fibrosis Research Advances
- Health Systems, Economic Evaluations, Quality of Life
- RNA Research and Splicing
- Muscle Physiology and Disorders
University of Michigan–Ann Arbor
2016-2025
Innovative BioTherapies (United States)
2024
Korea National Institute of Health
2024
Hudson Institute
2024
John Wiley & Sons (United States)
2024
University of North Dakota
2023
Michigan Medicine
2020-2022
University Health System
2020
Michigan United
2019
The Ohio State University Wexner Medical Center
2019
Sodium phenylbutyrate and taurursodiol have been found to reduce neuronal death in experimental models. The efficacy safety of a combination the two compounds persons with amyotrophic lateral sclerosis (ALS) are not known.
Abstract An orally administered, fixed‐dose coformulation of sodium phenylbutyrate‐taurursodiol (PB‐TURSO) significantly slowed functional decline in a randomized, placebo‐controlled, phase 2 trial ALS (CENTAUR). Herein we report results long‐term survival analysis participants CENTAUR. In CENTAUR, adults with were randomized 2:1 to PB‐TURSO or placebo. Participants completing the 6‐month (24‐week) eligible receive open‐label extension. all‐cause mortality (35‐month maximum follow‐up...
<h3>Importance</h3> Amyotrophic lateral sclerosis (ALS) has an immune component, but previous human studies have not examined changes over time. <h3>Objectives</h3> To assess peripheral inflammatory markers in participants with ALS and healthy control individuals to track determine whether these correlate disease progression. <h3>Design, Setting, Participants</h3> In this longitudinal cohort study, leukocytes were isolated from blood samples 35 controls 119 at the Clinic of University...
Persistent environmental pollutants may represent a modifiable risk factor involved in the gene-time-environment hypothesis amyotrophic lateral sclerosis (ALS).
To test the safety of spinal cord transplantation human stem cells in patients with amyotrophic lateral sclerosis (ALS) escalating doses and expansion trial to multiple clinical centers.This open-label included 15 participants at 3 academic centers divided into 5 treatment groups receiving increasing by numbers cells/injection injections. All received bilateral injections cervical (C3-C5). The final group both lumbar (L2-L4) through 2 separate surgical procedures. Participants were assessed...
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease of the motor nervous system. Clinical studies have demonstrated cortical and spinal neuron hyperexcitability using transcranial magnetic stimulation threshold tracking nerve conduction studies, respectively, although metrics excitability not been used as pharmacodynamic biomarkers in multi-site clinical trials.To ascertain whether ezogabine decreases ALS.This double-blind, placebo-controlled phase 2 randomized...
An interim report of a case-control study was conducted to explore the role environmental factors in development amyotrophic lateral sclerosis (ALS). Sixty-six cases and 66 age- gender-matched controls were recruited. Detailed information regarding residence history, occupational smoking, physical activity, other obtained using questionnaires. The association ALS with potential risk factors, including activity chemical exposure, investigated conditional logistic regression models. As...
SARM1, a protein with critical NADase activity, is central executioner in conserved programme of axon degeneration. We report seven rare missense or in-frame microdeletion human SARM1 variant alleles patients amyotrophic lateral sclerosis (ALS) other motor nerve disorders that alter the auto-inhibitory ARM domain and constitutively hyperactivate activity. The constitutive activity these variants similar to lacking entire greatly exceeds wild-type even presence nicotinamide mononucleotide...
Coformulated sodium phenylbutyrate/taurursodiol (PB/TURSO) was shown to prolong survival and slow functional decline in amyotrophic lateral sclerosis (ALS).
Objective Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by altered metabolome and energy homeostasis, manifesting with body mass index changes hypermetabolism—both prognostic of progression survival. The cross‐sectional ALS has been characterized, but longitudinal correlations to functional decline are lacking. Methods We longitudinally evaluated metabolomes from plasma terminal postmortem spinal cord brain motor cortex tissue. constructed 3 models. A...
To elucidate amyotrophic lateral sclerosis (ALS) biomarkers and potential mechanisms of disease, we measured immune cell populations in whole blood from a large cohort patients with ALS.Leukocytes were isolated the 44 control 90 ALS. The percentages total numbers each population analyzed using flow cytometry matched patient ALS Functional Rating Scale-Revised (ALSFRS-R) score to correlate leukocyte metrics disease progression.We show significant increase percentage neutrophils decrease CD4 T...
Objective To identify dysregulated metabolic pathways in amyotrophic lateral sclerosis (ALS) versus control participants through untargeted metabolomics. Methods Untargeted metabolomics was performed on plasma from ALS (n=125) around 6.8 months after diagnosis and healthy controls (n=71). Individual differential metabolites cases were assessed by Wilcoxon rank-sum tests, adjusted logistic regression partial least squares-discriminant analysis (PLS-DA), while group lasso explored...
Abstract Objective Intraspinal human spinal cord‐derived neural stem cell ( HSSC ) transplantation is a potential therapy for amyotrophic lateral sclerosis ALS ); however, previous trials lack controls. This post hoc analysis compared ambulatory limb‐onset participants in Phase 1 and 2 (Ph1/2) open‐label intraspinal studies up to 3 years after transplant matched Pooled Resource Open‐Access Clinical Trials PRO ‐ ACT ceftriaxone datasets provide required analyses inform future clinical trial...
To determine whether neutrophils contribute to amyotrophic lateral sclerosis (ALS) progression, we tested the association of baseline neutrophil count on ALS survival, effect was sex specific, and accumulate in spinal cord.A prospective cohort study conducted between June 22, 2011, October 30, 2019. Blood leukocytes were isolated from participants levels assessed by flow cytometry. Participant survival outcomes analyzed groups (<2 × 106, 2-4 >4 106 neutrophils/mL) with adjustments for...
Penalized regression methods are used in many biomedical applications for variable selection and simultaneous coefficient estimation. However, missing data complicates the implementation of these methods, particularly when missingness is handled using multiple imputation. Applying a algorithm on each imputed dataset will likely lead to different sets selected predictors. This paper considers general class penalized objective functions which, by construction, force same variables across...