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Understanding the Perseverance of the Muscular Dystrophy Community One-Year into the COVID-19 Pandemic.
- Published in:
- Journal of Neuromuscular Diseases, 2022, v. 9, n. 4, p. 517, doi. 10.3233/JND-220794
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- Publication type:
- Article
A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial.
- Published in:
- Journal of Neuromuscular Diseases, 2021, v. 8, n. 5, p. 769, doi. 10.3233/JND-210689
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- Publication type:
- Article
Upper Motor Neuron Disorders: Primary Lateral Sclerosis, Upper Motor Neuron Dominant Amyotrophic Lateral Sclerosis, and Hereditary Spastic Paraplegia.
- Published in:
- Brain Sciences (2076-3425), 2021, v. 11, n. 5, p. 611, doi. 10.3390/brainsci11050611
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- Publication type:
- Article
89 Two Newly Developed Frontiers CTSI Applications to Support Recruitment and Trial Management: The Frontiers Trial Finder Mobile App and a Predictive Accrual Web-based App.
- Published in:
- 2024
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- Publication type:
- Abstract
Mexiletine for Symptoms and Signs of Myotonia in Nondystrophic Myotonia: A Randomized Controlled Trial.
- Published in:
- JAMA: Journal of the American Medical Association, 2012, v. 308, n. 13, p. 1357, doi. 10.1001/jama.2012.12607
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- Publication type:
- Article
Autosomal dominant in cis D4Z4 repeat array duplication alleles in facioscapulohumeral dystrophy.
- Published in:
- Brain: A Journal of Neurology, 2024, v. 147, n. 2, p. 414, doi. 10.1093/brain/awad312
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- Publication type:
- Article
Reply: Wheelchair use in genetically confirmed FSHD1 from a large cohort study in Chinese population.
- Published in:
- 2022
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- Publication type:
- editorial
Predictors of functional outcomes in patients with facioscapulohumeral muscular dystrophy.
- Published in:
- 2021
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- Publication type:
- journal article
Machine learning suggests polygenic risk for cognitive dysfunction in amyotrophic lateral sclerosis.
- Published in:
- EMBO Molecular Medicine, 2021, v. 13, n. 1, p. 1, doi. 10.15252/emmm.202012595
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- Publication type:
- Article
Effect of Mexiletine on Muscle Stiffness in Patients With Nondystrophic Myotonia Evaluated Using Aggregated N-of-1 Trials.
- Published in:
- 2018
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- Publication type:
- journal article
Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study.
- Published in:
- 2019
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- Publication type:
- journal article
Using automated electronic medical record data extraction to model ALS survival and progression.
- Published in:
- 2018
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- Publication type:
- journal article
Muscarinic receptor activation tunes mouse stratum oriens interneurones to amplify spike reliability.
- Published in:
- Journal of Physiology, 2006, v. 571, n. 3, p. 555, doi. 10.1113/jphysiol.2005.103218
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- Publication type:
- Article
Cell type-specific dependence of muscarinic signalling in mouse hippocampal stratum oriens interneurones.
- Published in:
- Journal of Physiology, 2006, v. 570, n. 3, p. 595, doi. 10.1113/jphysiol.2005.100875
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- Publication type:
- Article
Correction to: A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot–Marie–Tooth type 1A.
- Published in:
- 2024
- By:
- Publication type:
- Correction Notice
Targeting protein homeostasis in sporadic inclusion body myositis.
- Published in:
- Science Translational Medicine, 2016, v. 8, n. 331, p. 1, doi. 10.1126/scitranslmed.aad4583
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- Publication type:
- Article
Combined N-of-1 trials to investigate mexiletine in non-dystrophic myotonia using a Bayesian approach; study rationale and protocol.
- Published in:
- BMC Neurology, 2015, v. 15, n. 1, p. 1, doi. 10.1186/s12883-015-0294-4
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- Publication type:
- Article
Combined N-of-1 trials to investigate mexiletine in non-dystrophic myotonia using a Bayesian approach; study rationale and protocol.
- Published in:
- 2015
- By:
- Publication type:
- journal article
Somatodendritic Kv7/KCNQ/M Channels Control Interspike Interval in Hippocampal Interneurons.
- Published in:
- Journal of Neuroscience, 2006, v. 26, n. 47, p. 9, doi. 10.1523/JNEUROSCI.3521-06.2006
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- Publication type:
- Article
Non-dystrophic myotonia: prospective study of objective and patient reported outcomes.
- Published in:
- 2013
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- Publication type:
- journal article
Non-dystrophic myotonia: prospective study of objective and patient reported outcomes.
- Published in:
- Brain: A Journal of Neurology, 2013, v. 136, n. 7, p. 2189
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- Publication type:
- Article
Using an onset-anchored Bayesian hierarchical model to improve predictions for amyotrophic lateral sclerosis disease progression.
- Published in:
- 2018
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- Publication type:
- journal article
Stabilization of Early Duchenne Cardiomyopathy With Aldosterone Inhibition: Results of the Multicenter AIDMD Trial.
- Published in:
- 2019
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- Publication type:
- journal article
Regional and bilateral MRI and gene signatures in facioscapulohumeral dystrophy: implications for clinical trial design and mechanisms of disease progression.
- Published in:
- Human Molecular Genetics, 2024, v. 33, n. 8, p. 698, doi. 10.1093/hmg/ddae007
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- Publication type:
- Article
Elevated plasma complement components in facioscapulohumeral dystrophy.
- Published in:
- Human Molecular Genetics, 2022, v. 31, n. 11, p. 1821, doi. 10.1093/hmg/ddab364
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- Publication type:
- Article
Phenotypic diversity in an international Cure VCP Disease registry.
- Published in:
- 2020
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- Publication type:
- journal article
Outcomes after intervention for enteral nutrition in patients with amyotrophic lateral sclerosis in multidisciplinary clinics.
- Published in:
- Muscle & Nerve, 2024, v. 70, n. 1, p. 94, doi. 10.1002/mus.28103
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- Publication type:
- Article
A longitudinal study of disease progression in facioscapulohumeral muscular dystrophy (FSHD).
- Published in:
- Muscle & Nerve, 2024, v. 69, n. 3, p. 362, doi. 10.1002/mus.28031
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- Publication type:
- Article
Self-reported reduced sleep quality and excessive daytime sleepiness in facioscapulohumeral muscular dystrophy.
- Published in:
- 2022
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- Publication type:
- journal article
Non‐dystrophic myotonia: 2‐year clinical and patient reported outcomes.
- Published in:
- Muscle & Nerve, 2022, v. 66, n. 2, p. 148, doi. 10.1002/mus.27649
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- Publication type:
- Article
Quantitative muscle analysis in facioscapulohumeral muscular dystrophy using whole‐body fat‐referenced MRI: Protocol development, multicenter feasibility, and repeatability.
- Published in:
- Muscle & Nerve, 2022, v. 66, n. 2, p. 183, doi. 10.1002/mus.27638
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- Publication type:
- Article
Open‐label pilot study of ranolazine for cramps in amyotrophic lateral sclerosis.
- Published in:
- Muscle & Nerve, 2022, v. 66, n. 1, p. 71, doi. 10.1002/mus.27560
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- Publication type:
- Article
Randomized phase 2 study of ACE‐083, a muscle‐promoting agent, in facioscapulohumeral muscular dystrophy.
- Published in:
- Muscle & Nerve, 2022, v. 66, n. 1, p. 50, doi. 10.1002/mus.27558
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- Publication type:
- Article
Brief assessment of cognitive function in myotonic dystrophy: Multicenter longitudinal study using computer‐assisted evaluation.
- Published in:
- Muscle & Nerve, 2022, v. 65, n. 5, p. 560, doi. 10.1002/mus.27520
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- Publication type:
- Article
Demographics, clinical characteristics, and prognostic factors of amyotrophic lateral sclerosis in Midwest.
- Published in:
- Muscle & Nerve, 2022, v. 65, n. 2, p. 217, doi. 10.1002/mus.27450
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- Publication type:
- Article
Long‐term efficacy and safety of dichlorphenamide for treatment of primary periodic paralysis.
- Published in:
- Muscle & Nerve, 2021, v. 64, n. 3, p. 342, doi. 10.1002/mus.27354
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- Publication type:
- Article
A patient‐focused survey to assess the effects of the COVID‐19 pandemic and social guidelines on people with muscular dystrophy.
- Published in:
- Muscle & Nerve, 2021, v. 64, n. 3, p. 321, doi. 10.1002/mus.27349
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- Publication type:
- Article
Guidelines on clinical presentation and management of nondystrophic myotonias.
- Published in:
- 2020
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- Publication type:
- journal article
Magnetic resonance imaging correlates with electrical impedance myography in facioscapulohumeral muscular dystrophy.
- Published in:
- 2020
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- Publication type:
- journal article
A pilot study of the responsiveness of wireless motion analysis in facioscapulohumeral muscular dystrophy.
- Published in:
- 2019
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- Publication type:
- journal article
Limb-girdle muscular dystrophy: A perspective from adult patients on what matters most.
- Published in:
- 2019
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- Publication type:
- journal article
Rasagiline for amyotrophic lateral sclerosis: A randomized, controlled trial.
- Published in:
- 2019
- By:
- Publication type:
- journal article
Electrical impedance myography in facioscapulohumeral muscular dystrophy: A 1-year follow-up study.
- Published in:
- 2018
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- Publication type:
- journal article
Facioscapulohumeral muscular dystrophy functional composite outcome measure.
- Published in:
- 2018
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- Publication type:
- journal article
Review of the Diagnosis and Treatment of Periodic Paralysis.
- Published in:
- 2018
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- Publication type:
- journal article
An instrumented timed up and go in facioscapulohumeral muscular dystrophy.
- Published in:
- 2018
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- Publication type:
- journal article
Validity of the 6 minute walk test in facioscapulohumeral muscular dystrophy.
- Published in:
- 2017
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- Publication type:
- journal article
Electrical impedance myography in facioscapulohumeral muscular dystrophy.
- Published in:
- 2016
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- Publication type:
- journal article
Muscle pathology grade for facioscapulohumeral muscular dystrophy biopsies.
- Published in:
- 2015
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- Publication type:
- journal article
Risk of functional impairment in Facioscapulohumeral muscular dystrophy.
- Published in:
- Muscle & Nerve, 2014, v. 49, n. 4, p. 520, doi. 10.1002/mus.23949
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- Publication type:
- Article