Found: 23
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Cerebellar transcranial magnetic stimulation impairs verbal working memory.
- Published in:
- Annals of Neurology, 2005, v. 58, n. 4, p. 553
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- Publication type:
- Article
Experience and Perspectives in the US on the Evolving Treatment Landscape in Spinal Muscular Atrophy.
- Published in:
- International Journal of General Medicine, 2022, v. 15, p. 7341, doi. 10.2147/IJGM.S369021
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- Publication type:
- Article
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial.
- Published in:
- 2018
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- Publication type:
- journal article
Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry.
- Published in:
- Journal of Neuromuscular Diseases, 2024, v. 11, n. 2, p. 425, doi. 10.3233/JND-230122
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- Publication type:
- Article
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG).
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- Journal of Neuromuscular Diseases, 2023, v. 10, n. 3, p. 389, doi. 10.3233/JND-221560
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- Publication type:
- Article
A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment.
- Published in:
- Journal of Neuromuscular Diseases, 2022, v. 9, n. 1, p. 39, doi. 10.3233/JND-210665
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- Publication type:
- Article
Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.
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- Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 989, doi. 10.3233/JND-210643
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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
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
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
Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double‐blind, placebo‐controlled, dose‐titration trial.
- Published in:
- Muscle & Nerve, 2021, v. 64, n. 3, p. 285, doi. 10.1002/mus.27347
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- Publication type:
- Article
Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study.
- Published in:
- Muscle & Nerve, 2021, v. 63, n. 5, p. 668, doi. 10.1002/mus.27187
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- Publication type:
- Article
The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID-19 pandemic.
- Published in:
- 2020
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- Publication type:
- journal article
Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials.
- Published in:
- 2020
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- Publication type:
- journal article
Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial.
- 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
Reverse fiber type disproportion: A distinct metabolic myopathy.
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- 2016
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- Publication type:
- journal article
Amifampridine phosphate (Firdapse(®)) is effective and safe in a phase 3 clinical trial in LEMS.
- Published in:
- 2016
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- Publication type:
- journal article
Mutations in the RNA exosome component gene EXOSC3 cause pontocerebellar hypoplasia and spinal motor neuron degeneration.
- Published in:
- Nature Genetics, 2012, v. 44, n. 6, p. 704, doi. 10.1038/ng.2254
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- Publication type:
- Article
Muscleblind-like 2 ( Mbnl2) -deficient mice as a model for myotonic dystrophy.
- Published in:
- Developmental Dynamics, 2008, v. 237, n. 2, p. 403, doi. 10.1002/dvdy.21428
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- Publication type:
- Article
Clinical Exome Sequencing for Genetic Identification of Rare Mendelian Disorders.
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- JAMA: Journal of the American Medical Association, 2014, v. 312, n. 18, p. 1880, doi. 10.1001/jama.2014.14604
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- Publication type:
- Article
Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
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- JAMA Neurology, 2022, v. 79, n. 10, p. 1005, doi. 10.1001/jamaneurol.2022.2480
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- Publication type:
- Article
Hearing and Vision Loss in an Older Man.
- Published in:
- JAMA Neurology, 2018, v. 75, n. 11, p. 1439, doi. 10.1001/jamaneurol.2018.1868
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- Publication type:
- Article