Early clinical and pre-clinical therapy development in Nemaline myopathy

Author:

Fisher Gemma1ORCID,Mackels Laurane12ORCID,Markati Theodora1ORCID,Sarkozy Anna3ORCID,Ochala Julien4ORCID,Jungbluth Heinz56ORCID,Ramdas Sithara17ORCID,Servais Laurent12ORCID

Affiliation:

1. MDUK Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK

2. Neuromuscular Reference Center, University and University Hospital of Liège, Liège, Belgium

3. Dubowitz Neuromuscular Centre, UCL Great Ormond Street Hospital, Institute of Child Health, London, UK

4. Department of Biomedical Sciences, University of Copenhagen, Copenhagen, Denmark

5. Department of Paediatric Neurology – Neuromuscular Service, Evelina Children’s Hospital, Guy’s & St Thomas’ NHS Foundation Trust, London, UK

6. Randall Centre for Cell and Molecular Biophysics, Muscle Signalling Section, Faculty of Life Sciences and Medicine (FoLSM), King’s College London, London, UK

7. Department of Paediatric Neurology, John Radcliffe Hospital, Oxford, UK

Publisher

Informa UK Limited

Subject

Clinical Biochemistry,Drug Discovery,Pharmacology,Molecular Medicine

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