St George's University of London.
Molecular & Clinical Sciences Research Institute,
Cell Biology and Genetics Research Centre,
St George's, University of London
Cranmer Terrace, London,
SW17 0RE, UK
Rare diseases, cell based therapies.
- Diagnosis delays, lack of comprehension of disease pathophysiology, poor co-ordination of care and no approved therapies are the challenges that face a majority of the rare disease community.
- Over 7,000 rare diseases have been identified, but to date there are only around 400 licenced treatments on the market, creating a huge unmet need.
- Currently her team are focusing on the fatal, ultra-rare disease, mitochondrial neurogastrointestinal encephalomyopathy (MNGIE).
Dr Bax’s research focuses on two objectives:
i) to improve the understanding of the underlying pathogenic molecular mechanisms of rare inherited diseases and
ii) to develop cell-based therapies for diseases with unmet needs.