Area of research
Neurology · Cellular and Molecular Neuroscience
Research interest
Research interests include Amyotrophic Lateral Sclerosis Research, Peripheral Neuropathies and Disorders, Genetic Neurodegenerative Diseases, and Neurogenetic and Muscular Disorders Research.
Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy
Phase <scp>2B</scp> randomized controlled trial of <scp>NP001</scp> in amyotrophic lateral sclerosis: Pre‐specified and post hoc analyses
Primary lateral sclerosis: consensus diagnostic criteria
Validation of serum neurofilaments as prognostic and potential pharmacodynamic biomarkers for ALS
Guidelines on clinical presentation and management of nondystrophic myotonias
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
Patient Assisted Intervention for Neuropathy: Comparison of Treatment in Real Life Situations (PAIN-CONTRoLS)
Preface: promoting research in PLS: current knowledge and future challenges
Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial
Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study
2017 European League Against Rheumatism/American College of Rheumatology classification criteria for adult and juvenile idiopathic inflammatory myopathies and their major subgroups
2017 European League Against Rheumatism/American College of Rheumatology Classification Criteria for Adult and Juvenile Idiopathic Inflammatory Myopathies and Their Major Subgroups
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
EULAR/ACR classification criteria for adult and juvenile idiopathic inflammatory myopathies and their major subgroups: a methodology report
A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy
Education and training of clinical and translational study investigators and research coordinators: A competency-based approach
A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease
Randomized, placebo-controlled trials of dichlorphenamide in periodic paralysis
Randomized phase 2 trial of NP001, a novel immune regulator
Ataluren treatment of patients with nonsense mutation dystrophinopathy
NP001 regulation of macrophage activation markers in ALS: A phase I clinical and biomarker study
Progress report on development of classification criteria for adult and juvenile idiopathic inflammatory myopathies
182. Progress Report on the Development of New Classification Criteria for Adult and Juvenile Idiopathic Inflammatory Myopathies
Deciphering amyotrophic lateral sclerosis: What phenotype, neuropathology and genetics are telling us about pathogenesis
OP0035 Progress Report on the Development of New Classification Criteria for Adult and Juvenile Idiopathic Inflammatory Myopathies
Progress report on the development of new classification criteria for adult and juvenile idiopathic inflammatory myopathies
Rituximab in the treatment of refractory adult and juvenile dermatomyositis and adult polymyositis: A randomized, placebo‐phase trial