UCL Great Ormond Street Institute of Child Health


Great Ormond Street Institute of Child Health

Dr Valeria Ricotti

Dr Valeria Ricotti

Honorary Lecturer

UCL GOS Institute of Child Health

Faculty of Pop Health Sciences

Joined UCL
1st Apr 2010

Research summary

Originally from Italy, I graduated with honours from RCSI, Ireland. In Dublin I completed my core paediatric training. At the beginning of 2010 I joined the Dubowitz Neuromuscular Centre, at the UCL Institute of Child Health and Great Ormond Street Hospital. I worked as a clinical researcher and investigator for numerous clinical trials for neuromuscular disorders, including the EU funded Skip-NMD project on antisense oligomer exon skipping (http://www.skip-nmd.eu), led by Professor Francesco Muntoni. I completed my higher degree focused on characterising the evolving natural history of Duchenne muscular dystrophy (DMD) and developing novel outcome measures for clinical trials. 


As part of my research projects I reported on the use, benefits and adverse effects of glucocorticoids in large cohorts of DMD subjects, and further delineated their cognitive and neurobehavioral profile in relation to their underlying genotype. I initiated collaborative efforts to deepen our understanding of the relationship between genotype and phenotype in DMD.  I have been an active member of international consortia, which developed new biomarkers and clinical outcome measures for neuromuscular disorders including activity monitors, myometers, functional scales and muscle MR imaging. I acted as a consultant for the Institute of Myology, University of Pierre et Marie Curie-Sorbonne, Paris.


With the goal to develop new pharmaco-gene therapies, I joined industry as Medical Director for the neuromuscular franchise of BioMarin Pharmaceutical Inc. Subsequently, I worked at Solid Biosciences as Director of Translational Research and Development, with a focus on the AAV-delivered micro-dystrophin gene therapy programme for DMD and the development of novel biomarkers.  

As an Honorary Lecturer at the Great Ormond Street Institute of Child Health I remain involved in academic research and notably in developing new technologies leveraging artificial intelligence and machine learning for monitoring neuromuscular diseases in children.


In April 2019 I co-founded DiNAQOR AG, a gene therapy biotech focused on advancing novel solutions for children and adults suffering from monogenic Cardiomyopathies (www.dinaqor.com). At DiNAQOR I currently hold a position as Executive Vice President and Chief Medical Officer. 

Teaching summary