Dr Valeria Ricotti
UCL GOS Institute of Child Health
Faculty of Pop Health Sciences
- Joined UCL
- 1st Apr 2010
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.