Using Natural History Studies for Rare Mitochondrial Diseases to Bolster Study Design
Time: 1:00 pm
day: Day Two
Details:
- Natural history studies are vital to all drug development projects but are especially difficult to obtain for inherited mitochondrial diseases due to their severity and rarity
- Discussing the power of natural history diseases to understand disease prevalence study disease progression and improve the efficiency of drug development
- Utilising natural history studies in conjunction to clinical trials due to the lack of alternate therapies as control treatment in rare mitochondrial diseases
