This case series has been developed with the assistance of Professor Jordi Díaz-Manera (John Walton Muscular Dystrophy Research Centre, Newcastle, UK), Dr. Cristina Domínguez González (Hospital Universitario 12 de Octubre, Madrid, Spain), and Dr. Federica Deodato (Ospedale Pediatrico Bambino Gesù, Rome, Italy). This HowITreat.MD activity was supported by Sanofi Genzyme and developed by SEI Healthcare.

The information presented is not intended as medical advice. Responsibility for patient care resides with the healthcare professional on the basis of their professional license, experience, and knowledge of the individual patient.

For full prescribing information, including indications, contraindications, warnings, precautions, and adverse events, please refer to the approved product labelling. Please note that products may have different product labelling according to geographical location. In Europe, full prescribing information is available from the European Medicines Agency (ema.europa.eu).

All characters and events depicted in this patient case simulation are entirely fictitious. Any similarity to actual events or persons, living or dead, is purely coincidental. This activity is intended for a global audience of healthcare professionals. To proceed, please confirm that you are a healthcare professional.