Richard Bedlack
A5102998448- Amyotrophic Lateral Sclerosis Research
- Neurogenetic and Muscular Disorders Research
- Parkinson's Disease Mechanisms and Treatments
- Prion Diseases and Protein Misfolding
- Cholinesterase and Neurodegenerative Diseases
- Genetic Neurodegenerative Diseases
- biodegradable polymer synthesis and properties
- Biochemical Acid Research Studies
- Health Systems, Economic Evaluations, Quality of Life
- Peripheral Neuropathies and Disorders
- Neurological diseases and metabolism
- Myasthenia Gravis and Thymoma
- Biomedical Ethics and Regulation
- Parkinson's Disease and Spinal Disorders
- Neurological disorders and treatments
- Neuroscience and Neural Engineering
- Pain Mechanisms and Treatments
- Alzheimer's disease research and treatments
- Histone Deacetylase Inhibitors Research
- Ion channel regulation and function
- Intracerebral and Subarachnoid Hemorrhage Research
- Adolescent and Pediatric Healthcare
- Synthetic Organic Chemistry Methods
- Cancer-related gene regulation
- Pesticide Exposure and Toxicity
Duke University
2016-2025
Duke Medical Center
2009-2024
Neurology, Inc
2023-2024
St. Jude Children's Research Hospital
2024
University of Miami
2024
Duke University Hospital
2001-2022
North Carolina State University
2022
Durham VA Medical Center
2008-2021
Perron Institute for Neurological and Translational Science
2020
The University of Western Australia
2020
Amyotrophic lateral sclerosis (ALS) is a devastating neurological disease with no effective treatment. We report the results of moderate-scale sequencing study aimed at increasing number genes known to contribute predisposition for ALS. performed whole-exome 2869 ALS patients and 6405 controls. Several were found be associated, TBK1 (the gene encoding TANK-binding kinase 1) was identified as an gene. bind phosphorylate proteins involved in innate immunity autophagy, including optineurin...
Amyotrophic lateral sclerosis (ALS) is the most common motor neuron disorder. While there are five FDA-approved drugs for treating this disease, each has only modest benefits. To design new and more effective therapies ALS, particularly sporadic ALS of unknown diverse etiologies, we must identify key, convergent mechanisms disease pathogenesis. This review focuses on origin effects glutamate-mediated excitotoxicity in (the cortical hyperexcitability hypothesis), which increased glutamatergic...
<h3>Objective</h3> To assess the reliability and usefulness of an EEG-based brain-computer interface (BCI) for patients with advanced amyotrophic lateral sclerosis (ALS) who used it independently at home up to 18 months. <h3>Methods</h3> Of 42 consented, 39 (93%) met study criteria, 37 (88%) were assessed use Wadsworth BCI. Nine (21%) could not other 28, 27 (men, age 28–79 years) (64%) had BCI placed in their homes, they caregivers trained it. Use data collected by Internet. Periodic visits...
The objective of the study was to establish safety and pharmacodynamics escalating dosages sodium phenylbutyrate (NaPB) in participants with ALS. Transcription dysregulation may play a role pathogenesis Sodium phenylbutyrate, histone deacetylase inhibitor, improves transcription post-transcriptional pathways, promoting cell survival mouse model motor neuron disease. Forty research at eight sites enrolled an open-label study. Study medication increased from 9 21 g/day. primary outcome measure...
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...
<h3>Objective:</h3> To determine the frequency of amyotrophic lateral sclerosis (ALS) plateaus and reversals in Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database. <h3>Methods:</h3> We analyzed Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) ALSFRS–revised (ALSFRS-R) data from PRO-ACT participants. The frequencies participants experiencing (periods where scores did not change) were calculated over 6-, 12-, 18-month epochs. percentage ever improved) different...
Importance The etiology of amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease, is unknown. However, neuroinflammation and complement activation may play role in disease progression. Objective To determine the effects zilucoplan, an inhibitor C5, individuals with ALS. Design, Setting, Participants Zilucoplan was tested as regimen A HEALEY ALS Platform Trial, phase 2 to 3 multicenter, randomized, double-blind, placebo-controlled perpetual platform clinical trial sharing...
We prospectively tested the quantitative myasthenia gravis score (QMG) for responsiveness and longitudinal construct validity in 53 patients with gravis. Index of was high. Longitudinal confirmed by correlation between changes QMG manual muscle testing a difference across that were clinically unchanged, improved, or worse two visits. Our results support use assessing clinical change trials.
Our objective was to determine the effect of creatine monohydrate on disease progression in patients with amyotrophic lateral sclerosis (ALS). One hundred and seven diagnosis probable or definite ALS, less than five years duration from symptom onset, were randomized either treatment daily (5 g/d) placebo. In this multicenter, double-blinded study we followed changes progression: using quantitative measures strength via maximal isometric voluntary contraction, forced vital capacity, ALSFRS,...
To assess the safety, tolerability, and preliminary efficacy of NP001, a novel immune regulator inflammatory monocytes/macrophages, for slowing progression amyotrophic lateral sclerosis (ALS).This was phase 2 randomized, double-blind, placebo-controlled trial NP001 in 136 patients with ALS <3 years' duration forced vital capacity ≥70%. Participants received mg/kg, 1 or placebo 6 months. Safety, biomarkers were assessed throughout study. Preliminary evaluated using Functional Rating...
To characterize the prevalence of cognitive and behavioral symptoms using a cognitive/behavioral screening battery in large prospective multicenter study amyotrophic lateral sclerosis (ALS).
<h3>Importance</h3> A new outcome measure for overall disability level with improved responsiveness is needed amyotrophic lateral sclerosis (ALS) clinical trials. <h3>Objective</h3> To describe the creation and development of a self-reported ALS scale item targeting psychometric properties that used mathematically rigorous Rasch methodology. <h3>Design, Setting, Participants</h3> preliminary questionnaire 119 questions was created based on literature review, judgement an expert panel,...
Abstract Coronavirus disease 2019 has created unprecedented challenges for amyotrophic lateral sclerosis (ALS) clinical care and research in the United States. Traditional evaluations making an ALS diagnosis, measuring progression, planning interventions rely on in‐person visits that may now be unsafe or impossible. Evidence‐ experience‐based treatment options, such as multidisciplinary team care, feeding tubes, wheelchairs, home health, hospice, have become more difficult to obtain some...
Objective There is a critical need to establish genetic markers that explain the complex phenotypes and pathogenicity of ALS. This study identified polymorphism in Stathmin-2 gene investigated its association with sporadic ALS (sALS) disease risk, age-of onset survival duration. Methods The candidate CA repeat was systematically analyzed using PCR, Sanger sequencing high throughput capillary separation for genotyping. expression RT-PCR patient olfactory neurosphere-derived (ONS) cells RNA...
Objective: Amyotrophic lateral sclerosis (ALS) has profound effects on people with ALS (PALS) and caregivers. There is a paucity of research detailing comparing PALS caregiver day-to-day perspectives ALS. Methods: A survey developed collaboratively by The Association panel experts in care was designed to broadly sample the experience caregivers respect physical emotional symptoms, efficacy treatment approaches, goals for future treatments. Specific symptoms assessed consisted fatigue, pain,...
ALS is a heterogeneous disease that may be complicated or in part driven by inflammation. NP001, regulator of macrophage activation, was associated with slowing progression those higher levels the plasma inflammatory marker C-reactive protein (CRP) phase 2A studies ALS. Here, we evaluate effects NP001 2B trial, and perform post hoc analysis combined data from preceding trial.The trial enrolled 138 participants within 3 y symptom onset hs-CRP values >1.13 mg/L. They were randomized 1:1 to...
This article reviews controversies surrounding the concept of myasthenic crisis. Literature review and our own experience demonstrate that there is disagreement about how a crisis should be defined, what implications are, whether crises are preventable, incidence calculated. We propose defined as weakness from acquired myasthenia gravis severe enough to necessitate intubation or delayed extubation following surgery. argue need not "doom" patient different prognosis response therapy compared...