Valeria Ricotti, MD, PhD | DuchenneXchange

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Researchers

Valeria Ricotti, MD, PhD

Researcher
Honorary Lecturer
Great Ormond Street Institute of Child Health
University College London
30 Guilford Street
London, United Kingdom

Dr Valeria Ricotti, originally from Italy, graduated with honours from RCSI, Ireland. She completed her core paediatric training in Dublin. Currently, she is the Director of Translational Research and Development at Solid Biosciences and involved in designing the AAV-delivered micro-dystrophin gene therapy clinical trial for DMD and developing clinical outcome measures. She 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 led by Professor Francesco Muntoni.

She completed her 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 her research projects, she 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. As an Honorary Lecturer at the Great Ormond Street Institute of Child Health, she remains involved in academic research and notably in developing new treatment approaches for neuromuscular diseases in children.

 

Representative Publications:

Semi-Automated Analysis of Diaphragmatic Motion with Dynamic Magnetic Resonance Imaging in Healthy Controls and Non-Ambulant Subjects with Duchenne Muscular Dystrophy

Outcome measures for Duchenne muscular dystrophy from ambulant to non-ambulant patients: implications for clinical trials

Quantification of microdystrophin and correlation to circulating biomarkers

Severe persistent injection site reactions after subcutaneous 2′-O-methyl phosphorothioate oligonucleotide therapy for Duchenne muscular dystrophy

SGT-001 Micro-dystrophin gene therapy for Duchenne muscular dystrophy