Development of a functional composite for the evaluation of spinal and bulbar muscular atrophy

[1]  K. Fischbeck,et al.  Disease mechanism, biomarker and therapeutics for spinal and bulbar muscular atrophy (SBMA) , 2020, Journal of Neurology, Neurosurgery, and Psychiatry.

[2]  Stephanie J. C. Taylor,et al.  Analysis of baseline data , 2018 .

[3]  G. Sobue,et al.  Treatment with Creatine Monohydrate in Spinal and Bulbar Muscular Atrophy: Protocol for a Randomized, Double-Blind, Placebo-Controlled Trial , 2018, JMIR research protocols.

[4]  G. Sobue,et al.  Long-term treatment with leuprorelin for spinal and bulbar muscular atrophy , 2017, Journal of the Neurological Sciences.

[5]  G. Sobue,et al.  Decreased Peak Expiratory Flow Associated with Muscle Fiber-Type Switching in Spinal and Bulbar Muscular Atrophy , 2016, PloS one.

[6]  Jeffrey A. Cohen,et al.  Alemtuzumab improves preexisting disability in active relapsing-remitting MS patients , 2016, Neurology.

[7]  G. Sobue,et al.  Head Lift Exercise Improves Swallowing Dysfunction in Spinal and Bulbar Muscular Atrophy , 2015, European Neurology.

[8]  T. Gheno,et al.  Planning future clinical trials in Machado Joseph disease: Lessons from a phase 2 trial , 2015, Journal of the Neurological Sciences.

[9]  K. Fischbeck,et al.  A functional scale for spinal and bulbar muscular atrophy: Cross-sectional and longitudinal study , 2015, Neuromuscular Disorders.

[10]  Veronica Redaelli,et al.  Presymptomatic cognitive and neuroanatomical changes in genetic frontotemporal dementia in the Genetic Frontotemporal dementia Initiative (GENFI) study: a cross-sectional analysis , 2015, The Lancet Neurology.

[11]  Shuling Guo,et al.  Peripheral androgen receptor gene suppression rescues disease in mouse models of spinal and bulbar muscular atrophy. , 2014, Cell reports.

[12]  K. Fischbeck,et al.  Assessing Function and Endurance in Adults with Spinal and Bulbar Muscular Atrophy: Validity of the Adult Myopathy Assessment Tool , 2014, Rehabilitation research and practice.

[13]  G. Sobue,et al.  Tongue pressure as a novel biomarker of spinal and bulbar muscular atrophy , 2014, Neurology.

[14]  Thomas Kohlmann,et al.  Systematic literature review and validity evaluation of the Expanded Disability Status Scale (EDSS) and the Multiple Sclerosis Functional Composite (MSFC) in patients with multiple sclerosis , 2013, BMC Neurology.

[15]  Alexandra Durr,et al.  Biological and clinical characteristics of individuals at risk for spinocerebellar ataxia types 1, 2, 3, and 6 in the longitudinal RISCA study: analysis of baseline data , 2013, The Lancet Neurology.

[16]  Chris Frost,et al.  Predictors of phenotypic progression and disease onset in premanifest and early-stage Huntington's disease in the TRACK-HD study: analysis of 36-month observational data , 2013, The Lancet Neurology.

[17]  C. Angelini,et al.  Pilot trial of clenbuterol in spinal and bulbar muscular atrophy , 2013, Neurology.

[18]  K. Abe,et al.  Assessment of swallowing in motor neuron disease and Asidan/SCA36 patients with new methods , 2013, Journal of the Neurological Sciences.

[19]  Y. Kanda,et al.  Investigation of the freely available easy-to-use software ‘EZR' for medical statistics , 2012, Bone Marrow Transplantation.

[20]  G. Sobue,et al.  Longitudinal changes of outcome measures in spinal and bulbar muscular atrophy. , 2012, Brain : a journal of neurology.

[21]  Nick C Fox,et al.  Clinical and biomarker changes in dominantly inherited Alzheimer's disease. , 2012, The New England journal of medicine.

[22]  G. Sobue,et al.  Difference in chronological changes of outcome measures between untreated and placebo-treated patients of spinal and bulbar muscular atrophy , 2012, Journal of Neurology.

[23]  V. de Groot,et al.  Which walking capacity tests to use in multiple sclerosis? A multicentre study providing the basis for a core set , 2012, Multiple sclerosis.

[24]  E. Wilson,et al.  An Interdomain Interaction of the Androgen Receptor Is Required for Its Aggregation and Toxicity in Spinal and Bulbar Muscular Atrophy* , 2010, The Journal of Biological Chemistry.

[25]  G. Sobue,et al.  The profile of motor unit number estimation (MUNE) in spinal and bulbar muscular atrophy , 2009, Journal of Neurology, Neurosurgery & Psychiatry.

[26]  K. Fischbeck,et al.  Clinical features of spinal and bulbar muscular atrophy , 2009, Brain : a journal of neurology.

[27]  E BERGLUND,et al.  Spirometric studies in normal subjects. I. Forced expirograms in subjects between 7 and 70 years of age. , 2009, Acta medica Scandinavica.

[28]  G. Sobue,et al.  Phase 2 trial of leuprorelin in patients with spinal and bulbar muscular atrophy , 2009, Annals of neurology.

[29]  D. Timmann,et al.  SCA Functional Index , 2008, Neurology.

[30]  G. Sobue,et al.  Walking capacity evaluated by the 6‐minute walk test in spinal and bulbar muscular atrophy , 2008, Muscle & nerve.

[31]  M. Ruberg,et al.  Composite cerebellar functional severity score: validation of a quantitative score of cerebellar impairment. , 2008, Brain : a journal of neurology.

[32]  Lippincott Williams Wilkins,et al.  Scale for the assessment and rating of ataxia: Development of a new clinical scale , 2006, Neurology.

[33]  G. Sobue,et al.  Pathogenesis, animal models and therapeutics in Spinal and bulbar muscular atrophy (SBMA) , 2006, Experimental Neurology.

[34]  A. Durr,et al.  Scale for the Assessment and Rating of Ataxia (SARA) , 2010 .

[35]  G. Sobue,et al.  Natural history of spinal and bulbar muscular atrophy (SBMA): a study of 223 Japanese patients. , 2006, Brain : a journal of neurology.

[36]  Y. Itoyama,et al.  [Study of functional rating scale for amyotrophic lateral sclerosis: revised ALSFRS(ALSFRS-R) Japanese version]. , 2001, No to shinkei = Brain and nerve.

[37]  J. Moncrieff,et al.  A multicentre, randomized, double-blind, placebo-controlled trial of naltrexone in the treatment of alcohol dependence or abuse. , 2000, Alcohol and alcoholism.

[38]  S. Reingold,et al.  The Multiple Sclerosis Functional Composite measure (MSFC): an integrated approach to MS clinical outcome assessment , 1999, Multiple sclerosis.

[39]  Michelle Mendoza,et al.  Guidelines for the use and performance of quantitative outcome measures in ALS clinical trials , 1997, Journal of the Neurological Sciences.

[40]  K. Fischbeck,et al.  Androgen receptor gene mutations in X-linked spinal and bulbar muscular atrophy , 1991, Nature.

[41]  Lewis E. Kazis,et al.  Effect Sizes for Interpreting Changes in Health Status , 1989, Medical care.

[42]  M Alter,et al.  Progressive proximal spinal and bulbar muscular atrophy of late onset. A sex-linked recessive trait. , 1968, Neurology.

[43]  D. Richards,et al.  Pulmonary in-sufficiency I. Physiological classification, clinical methods of analysis, standard values in normal subjects , 1948 .

[44]  Michelle Marie Tipton-Burton.  Amyotrophic Lateral Sclerosis Functional Rating Scale , 2017 .

[45]  F. Islami,et al.  Multimorbidity: Epidemiology and Risk Factors in the Golestan Cohort Study, Iran , 2016, Medicine.

[46]  Abdulmajeed Al Abdukareem.  Randomized, placebo-controlled trial , 2004, Annals of Saudi medicine.

[47]  A. Pestronk,et al.  Assessment of disease progression in dysferlinopathy A 1-year cohort study , 2022 .