About the Authors

Palittiya Sintusek

Affiliations Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, London, United Kingdom, Division of Gastroenterology and Hepatology, Department of Pediatrics, Faculty of Medicine, King Chulalongkorn Memorial Hospital, Chulalongkorn University, Bangkok, Thailand

Francesco Catapano

Affiliation Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, London, United Kingdom

Napat Angkathunkayul

Affiliation Department of Pathology, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand

Elena Marrosu

Affiliation Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, London, United Kingdom

Simon H. Parson

Affiliations Institute of Medical Sciences, University of Aberdeen, Foresterhill, Aberdeen, United Kingdom, Euan MacDonald Center for Motor Neuron Disease Research, University of Edinburgh, Edinburgh, United Kingdom

Jennifer E. Morgan

Affiliation Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, London, United Kingdom

Francesco Muntoni

Haiyan.zhou@ucl.ac.uk (HZ); f.muntoni@ucl.ac.uk (FM)

Affiliation Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, London, United Kingdom

Haiyan Zhou

Haiyan.zhou@ucl.ac.uk (HZ); f.muntoni@ucl.ac.uk (FM)

Affiliation Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, London, United Kingdom

Competing Interests

FM is involved as a principal investigator in SMA clinical trials in collaboration with Ionis and Biogen, and Roche. He is also member of the Scientific Advisory Board of the Rare Disease section at Pfizer. This does not alter our adherence to PLOS ONE policies on sharing data and materials. The remaining authors disclose no conflicts.

Author Contributions

Conceived and designed the experiments: HZ FM. Performed the experiments: HZ PS FC EM. Analyzed the data: HZ PS NA. Wrote the paper: HZ PS JEM SHP FM.