Bridget Bax

• 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.