Michael Benatar
A5031062952- Amyotrophic Lateral Sclerosis Research
- Neurogenetic and Muscular Disorders Research
- Myasthenia Gravis and Thymoma
- Peripheral Neuropathies and Disorders
- Parkinson's Disease Mechanisms and Treatments
- Genetic Neurodegenerative Diseases
- Parkinson's Disease and Spinal Disorders
- Prion Diseases and Protein Misfolding
- Neurological diseases and metabolism
- Muscle Physiology and Disorders
- Neurological disorders and treatments
- Peripheral Nerve Disorders
- Antifungal resistance and susceptibility
- Hereditary Neurological Disorders
- Alzheimer's disease research and treatments
- Botulinum Toxin and Related Neurological Disorders
- Health Systems, Economic Evaluations, Quality of Life
- Cardiomyopathy and Myosin Studies
- Cholinesterase and Neurodegenerative Diseases
- Metabolism and Genetic Disorders
- Mitochondrial Function and Pathology
- Muscle activation and electromyography studies
- Neurological and metabolic disorders
- Autoimmune Neurological Disorders and Treatments
- Cancer Treatment and Pharmacology
University of Miami
2016-2025
St. Jude Children's Research Hospital
2013-2024
Duke University
2017-2024
The University of Texas Southwestern Medical Center
2016-2023
Emory University
2004-2023
Jacksonville College
2022-2023
Mayo Clinic in Florida
2017-2023
VIB-UAntwerp Center for Molecular Neurology
2022-2023
University of Antwerp
2022-2023
Amyotrophic Lateral Sclerosis Association
2023
Thymectomy has been a mainstay in the treatment of myasthenia gravis, but there is no conclusive evidence its benefit. We conducted multicenter, randomized trial comparing thymectomy plus prednisone with alone.We compared extended transsternal alternate-day alone. Patients 18 to 65 years age who had generalized nonthymomatous gravis disease duration less than 5 were included if they Myasthenia Gravis Foundation America clinical class II IV (on scale from I V, higher classes indicating more...
The intrathecally administered antisense oligonucleotide tofersen reduces synthesis of the superoxide dismutase 1 (SOD1) protein and is being studied in patients with amyotrophic lateral sclerosis (ALS) associated mutations SOD1 (SOD1 ALS).
Poly(GP) proteins are a promising pharmacodynamic marker for developing and testing therapeutics treating C9ORF72 -associated amyotrophic lateral sclerosis.
Objective To evaluate neurofilament light (NfL) as a biomarker of the presymptomatic phase amyotrophic lateral sclerosis (ALS). Methods The study population includes 84 individuals at risk for developing ALS, 34 controls, 17 ALS patients, and 10 phenoconverters (at‐risk observed both before after emergence clinically manifest disease). At‐risk are enrolled through Pre‐Symptomatic Familial ( Pre‐fALS ), longitudinal natural history who carriers any ALS‐associated gene mutation (in SOD1,...
To evaluate the efficacy of rituximab in treatment anti-muscle-specific kinase (MuSK) myasthenia gravis (MG).This was a multicenter, blinded, prospective review, comparing anti-MuSK-positive patients with MG treated to those not rituximab. The primary clinical endpoint Myasthenia Gravis Status and Treatment Intensity (MGSTI), novel outcome that combines Foundation America (MGFA) postintervention status (PIS) number dosages other immunosuppressant therapies used. A priori, an MGSTI level ≤2...
Complement activation at the neuromuscular junction is a primary cause of acetylcholine receptor loss and failure transmission in myasthenia gravis (MG). Eculizumab, humanized monoclonal antibody, blocks formation terminal complement complex by specifically preventing enzymatic cleavage 5 (C5).This study was randomized, double-blind, placebo-controlled, crossover trial involving 14 patients with severe, refractory generalized MG (gMG).Six 7 treated eculizumab for 16 weeks (86%) achieved...
Eculizumab is effective and well tolerated in patients with antiacetylcholine receptor antibody-positive refractory generalized myasthenia gravis (gMG; REGAIN; NCT01997229). We report an interim analysis of open-label extension REGAIN, evaluating eculizumab's long-term safety efficacy.Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients.The profile eculizumab consistent no cases meningococcal infection were reported during the period. Myasthenia...
To develop recommendations for the evaluation, diagnosis, prognostication, and treatment of facioscapulohumeral muscular dystrophy (FSHD) from a systematic review analysis evidence.Relevant articles were analyzed in accordance with American Academy Neurology classification evidence schemes diagnostic, prognostic, studies. Recommendations linked to strength other factors.Available genetic testing FSHD type 1 is highly sensitive specific. Although respiratory insufficiency occurs rarely FSHD,...
To identify preferred neurofilament assays and clinically validate serum light (NfL) phosphorylated heavy (pNfH) as prognostic potential pharmacodynamic biomarkers relevant to amyotrophic lateral sclerosis (ALS) therapy development.In this prospective, multicenter, longitudinal observational study of patients with ALS (n = 229), primary 20), progressive muscular atrophy 11), biological specimens were collected, processed, stored according strict standard operating procedures (SOPs)....
<h3>Importance</h3> Many patients with generalized myasthenia gravis (gMG) have substantial clinical disability, persistent disease burden, and adverse effects attributable to chronic immunosuppression. Therefore, there is a significant need for targeted, well-tolerated therapies the potential improve control enhance quality of life. <h3>Objective</h3> To evaluate zilucoplan, subcutaneously (SC) self-administered macrocyclic peptide inhibitor complement component 5, in broad population...
Despite extensive research, amyotrophic lateral sclerosis (ALS) remains a progressive and invariably fatal neurodegenerative disease. Limited knowledge of the underlying causes ALS has made it difficult to target upstream biological mechanisms disease, therapeutic interventions are usually administered relatively late in course Genetic forms offer unique opportunity for development, as genetic associations may reveal potential insights into disease etiology. also be amenable investigating...
Significance Our study, involving 1,873 patients and 36,370 healthy individuals, is an extensive genome-wide study of myasthenia gravis. association transcriptome-wide analyses identified two signals, namely CHRNA1 CHRNB1 , encoding acetylcholine receptor subunits, which were replicated in independent cohort obtained from the UK Biobank. Identifying these genes confirms potential utility using genetics to identify proteins that are antigenic targets autoantibodies. We confirmed genetic...