Lessons Learned From Translational Research in Neuromuscular Diseases: Impact on Study Design, Outcome Measures and Managing Expectation

• Georgia Stimpson,
• Mary Chesshyre,
• Mary Chesshyre,
• Giovanni Baranello,
• Giovanni Baranello,
• Francesco Muntoni,
• Francesco Muntoni

Affiliations

Georgia Stimpson

Developmental Neuroscience Research and Training Department, Dubowitz Neuromuscular Centre, Faculty of Population Health Sciences, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom

Mary Chesshyre

Developmental Neuroscience Research and Training Department, Dubowitz Neuromuscular Centre, Faculty of Population Health Sciences, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom

Mary Chesshyre

NIHR Great Ormond Street Hospital Biomedical Research Centre, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom

Giovanni Baranello

Developmental Neuroscience Research and Training Department, Dubowitz Neuromuscular Centre, Faculty of Population Health Sciences, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom

Giovanni Baranello

NIHR Great Ormond Street Hospital Biomedical Research Centre, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom

Francesco Muntoni

Developmental Neuroscience Research and Training Department, Dubowitz Neuromuscular Centre, Faculty of Population Health Sciences, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom

Francesco Muntoni

NIHR Great Ormond Street Hospital Biomedical Research Centre, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom