A5102925672- Amyotrophic Lateral Sclerosis Research
- Neurogenetic and Muscular Disorders Research
- Parkinson's Disease Mechanisms and Treatments
- Myasthenia Gravis and Thymoma
- Neurological diseases and metabolism
- Peripheral Neuropathies and Disorders
- Genetic Neurodegenerative Diseases
- Prion Diseases and Protein Misfolding
- Parkinson's Disease and Spinal Disorders
- Cholinesterase and Neurodegenerative Diseases
- Alzheimer's disease research and treatments
- Nerve injury and regeneration
- Pharmaceutical studies and practices
- Endoplasmic Reticulum Stress and Disease
- Neurological disorders and treatments
- Hereditary Neurological Disorders
- Synthetic Organic Chemistry Methods
- Head and Neck Surgical Oncology
- CNS Lymphoma Diagnosis and Treatment
- Healthcare Decision-Making and Restraints
- Alcoholism and Thiamine Deficiency
- Coordination Chemistry and Organometallics
- RNA Research and Splicing
- Muscle Physiology and Disorders
- Ocular Oncology and Treatments
Loma Linda University
2017-2025
Loma Linda University Medical Center
2018-2024
University of Kansas Medical Center
2016-2019
University of Miami
2016-2019
Massachusetts General Hospital
2003-2019
Carolinas Medical Center
2001-2019
University of California, San Francisco
2008-2019
Harvard University
2003-2019
Mayo Clinic in Arizona
2019
University Medical Center Utrecht
2019
Approximately 90% of persons with amyotrophic lateral sclerosis (ALS) have the sporadic form, which may be caused by interaction multiple environmental factors and previously unknown genes.We performed a genomewide association analysis using 766,955 single-nucleotide polymorphisms (SNPs) found in 386 white patients ALS 542 neurologically normal controls (the discovery series). Associations SNPs were confirmed two independent replication populations: series 1, 766 case disease 750 controls,...
<b><i>Objective:</i></b> To determine if long-term topiramate therapy is safe and slows disease progression in patients with ALS. <b><i>Methods:</i></b> A double-blind, placebo-controlled, multicenter randomized clinical trial was conducted. Participants ALS (n = 296) were (2:1) to receive (maximum tolerated dose up 800 mg/day) or placebo for 12 months. The primary outcome measure the rate of change upper extremity motor function as measured by maximum voluntary isometric contraction (MVIC)...
Abstract Twenty ALS patients with sialorrhea refractory to medical therapy were enrolled in this double‐blind, randomized study receive either 2,500 U of botulinum toxin type B (BTxb) or placebo into the bilateral parotid and submandibular glands using electromyographic guidance. Patients who received BTxb reported a global impression improvement 82% at 2 weeks compared 38% those ( P < 0.05). This significant effect was sustained 4 weeks. At 12 weeks, 50% continued report 14% placebo....
To revise the 1999 Airlie House consensus guidelines for design and implementation of preclinical therapeutic studies clinical trials in amyotrophic lateral sclerosis (ALS).A committee comprising 140 key members international ALS community (ALS researchers, clinicians, patient representatives, research funding industry, regulatory agencies) addressed 9 areas need within research: (1) studies; (2) biological phenotypic heterogeneity; (3) outcome measures; (4) disease-modifying symptomatic...
To determine the steroid-sparing effect of methotrexate (MTX) in patients with symptomatic generalized myasthenia gravis (MG).We performed a 12-month multicenter, randomized, double-blind, placebo-controlled trial MTX 20 mg orally every week vs placebo 50 acetylcholine receptor antibody-positive MG between April 2009 and August 2014. The primary outcome measure was prednisone area under dose-time curve (AUDTC) from months 4 to 12. Secondary measures included changes Quantitative Myasthenia...
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...
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,...
Objectives Ceftriaxone increases expression of the astrocytic glutamate transporter, EAAT2, which might protect from glutamate-mediated excitotoxicity. A trial using a novel three stage nonstop design, incorporating Phases I-III, tested ceftriaxone in ALS. Stage 1 determined cerebrospinal fluid pharmacokinetics subjects with 2 evaluated safety and tolerability for 20-weeks. Analysis pharmacokinetics, tolerability, was used to determine dosage 3 efficacy testing. Methods In 1, 66 at ten...
OBJECTIVE: To compare characteristics of ALS patients with and without percutaneous endoscopic gastrostomy (PEG).METHODS: Using the Patient Care Database, data from PEG Functional Rating Scale‐bulbar subscale (ALSFRSb) scores ≤5 were analyzed; follow‐up also collected.RESULTS: use was markedly increased declining ALSFRSb scores. Demographics did not differ, but ALSFRS composite bulbar arm lower (P<0.0001). used significantly more assistive devices, multidisciplinary care, home care nurses...
Abstract Assembly modulators are a new class of allosteric site-targeted therapeutic small molecules, some which effective at restoring nuclear localization TDP-43 in ALS cellular models, and display efficacy variety animal models. These compounds have been shown to bind selectively subset protein disulfide isomerase (PDI), implicated pathophysiology. The targeted PDI is found within novel, transient energy-dependent multi-protein complex that includes other important members the...