A5014335916- Amyotrophic Lateral Sclerosis Research
- Neurogenetic and Muscular Disorders Research
- Parkinson's Disease Mechanisms and Treatments
- Muscle activation and electromyography studies
- Prion Diseases and Protein Misfolding
- Neuroscience and Neural Engineering
- Neurological disorders and treatments
- Cholinesterase and Neurodegenerative Diseases
- Peripheral Nerve Disorders
- Genetic Neurodegenerative Diseases
- biodegradable polymer synthesis and properties
- Nerve injury and regeneration
- Peripheral Neuropathies and Disorders
- Biochemical Acid Research Studies
- Neurological diseases and metabolism
- Synthetic Organic Chemistry Methods
- Spinal Dysraphism and Malformations
- Transcranial Magnetic Stimulation Studies
- Neural dynamics and brain function
- Botulinum Toxin and Related Neurological Disorders
- Hereditary Neurological Disorders
- Dysphagia Assessment and Management
- Visual perception and processing mechanisms
- Spinal Cord Injury Research
- Retinal Development and Disorders
Barrow Neurological Institute
2016-2025
University of Arizona
2016-2025
Creighton University
2019-2025
Phoenix (United States)
2020-2023
Massachusetts General Hospital
1999-2023
CREATe Centre
2023
Harvard University
1995-2023
Case Western Reserve University
2023
St. Joseph's Hospital and Medical Center
2015-2021
Montreal Neurological Institute and Hospital
2020
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.
© 2020 The Authors. Published by Elsevier B.V. on behalf of International Federation Clinical Neurophysiology. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
Abstract When a peripheral nerve is severed and left untreated, the most likely result formation of an endbulb neuroma; this tangled mass disorganized fibers blocks functional recovery following injury. Although there are several different approaches for promoting repair, which have been greatly refined over recent years, clinical results repair remain very disappointing. In paper we compare collagen guide conduit to more standard procedure autografting promote transected nerves in rats...
<h3>Objective</h3> To report results of intrathecal nusinersen in children with later-onset spinal muscular atrophy (SMA). <h3>Methods</h3> Analyses included from a phase 1b/2a study (ISIS-396443-CS2; NCT01703988) who first received during that and were eligible to continue treatment the extension (ISIS-396443-CS12; NCT02052791). The was 253-day, ascending dose (3, 6, 9, 12 mg), multiple-dose, open-label, multicenter enrolled SMA aged 2–15 years. 715-day, single-dose level (12 mg) study....
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...
Objective The diagnosis of amyotrophic lateral sclerosis (ALS) remains problematic, with current diagnostic criteria (revised El Escorial [rEEC] and Awaji) being complex prone to error. Consequently, the utility recently proposed Gold Coast was determined in ALS. Methods We retrospectively reviewed 506 patients (302 males, 204 females) compare accuracy that Awaji rEEC (defined by proportion categorized as definite, probable, or possible ALS) accordance standards reporting criteria. Results...
Nerve regeneration was followed in 15 median and 1 ulnar nerve of eight Macaca fascicularis monkeys by serial electrophysiological assessments over a period three half years. gaps 5 mm at the wrist were bridged collagen-based guides, autografts, or direct suture repairs. Thenar muscle reinnervation occurred between 50 70 d for all groups, indicating axonal elongation rates approximately mm/d. The recovery compound action potential (CMAP) sensory (CSAP) amplitudes significantly slower after...
Abstract Objective To determine whether chronic treatment with celecoxib, a cyclooxygenase‐2 inhibitor that has been shown to be beneficial in preclinical testing, is safe and effective amyotrophic lateral sclerosis (ALS). Methods A double‐blind, placebo‐controlled, clinical trial was conducted. Three hundred research subjects ALS were randomized (2:1) receive celecoxib (800mg/day) or placebo for 12 months. The primary outcome measure the rate of change upper extremity motor function...
<b><i>Background:</i></b> Mitochondrial dysfunction occurs early in the course of ALS, and mitochondria may be an important site for therapeutic intervention. Creatine stabilizes mitochondrial transition pore, is ATP production. In a transgenic mouse model administration creatine prolongs survival preserves motor function neurons. <b><i>Methods:</i></b> The authors conducted randomized double-blind, placebo controlled trial on 104 patients with ALS from 14 sites to evaluate efficacy...
Abstract Objective We explored the potential of embryonic stem cell–derived motor neurons to functionally replace those cells destroyed in paralyzed adult rats. Methods administered a phosphodiesterase type 4 inhibitor and dibutyryl cyclic adenosine monophosphate overcome myelin‐mediated repulsion provided glial neurotrophic factor within sciatic nerve attract transplanted axons toward skeletal muscle targets. Results found that these strategies significantly increased success extending out...
Abstract Objective Amyotrophic lateral sclerosis (ALS) is a devastating, and currently incurable, neuromuscular disease in which oxidative stress mitochondrial impairment are contributing to neuronal loss. Coenzyme Q10 (CoQ10), an antioxidant cofactor, has shown promise ALS transgenic mice, clinical trials for neurodegenerative diseases other than ALS. Our aims were choose between two high doses of CoQ10 ALS, determine if it merits testing Phase III trial. Methods We designed implemented...
<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)...
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...