Originally from Italy, Dr Mariacristina Scoto is a consultant in neuromuscular translational research at Great Ormond Street Hospital for Children and is honorary associate professor at UCL Great Ormond Street Institute of Child Health, London. She is principal investigator (PI) and co-PI of a number of clinical trials, in both Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). Her main interest is on the evolving natural history and standard of care of spinal muscular atrophy with the advent of innovative treatments, and is Co-PI of the SMA REACH (Research and Clinical Hub) UK Network.