Theme Lead: Professor Francesco Muntoni
At Great Ormond Street Hospital (GOSH) and the UCL Institute of Child Health (ICH) we see many rare childhood diseases. Our unique access to groups of patients suffering from similar, rare diseases allows us to develop and evaluate novel therapies and treatments.
At the core of our research is the definition of clinical syndromes and the design and testing of innovative interventions.
Our key research topics highlight the diversity of our work into novel therapeutics:
- Managing heart disease though use of percutaneous catheters
- Improving the evaluation and management of epilepsy
- The neurochemistry of epilepsy
- Improving the understanding of childhood stroke and evaluating novel interventions to aid prognosis
- Novel approaches to treating muscle disease
- Prevention of spinal cord defects
- Use of enteric nervous system stem cells as a potential treatment for severe gut developmental disorders
- Novel cancer cell treatments
- Treating rheumatological disease of childhood
- Evaluating new strategies for preventing infections in hospitalised settings