Mitochondrial disorders have emerged as a major cause of inherited human disease. Although the past decade has seen major advances in our understanding of their genetic basis and the underlying pathology, these findings have yet to translate into new therapies. There is a growing appreciation that new treatments will only emerge through a concerted collaboration between clinicians, laboratory scientists and the life sciences industry, based on a firm understanding of the disease mechanisms.
This conference will build new partnerships that harness our understanding of the disease mechanisms, accelerating the pace of effective treatments for mitochondrial diseases.
Topics will include:
- Mitochondrial medicine – pharma and funding perspectives
- Pre-clinical mitochondrial disease models
- Clinical trials
- Informatics and data sharing to support precision medicine
- Regulatory guidance for therapeutic development
Virtual networking sessions will be included.