Rare disease awareness

What happened: Nasha Fitter, a tech entrepreneur, is advocating for rare disease awareness after her daughter was diagnosed with FOXG1 syndrome in 2017. CNBC Cures, a platform launched last summer, aims to spotlight the 30 million Americans living with a rare disease. Every Cure, a biotech company, is pursuing a drug repurposing strategy for rare diseases, targeting the 95% without cures due to high development costs and small patient populations.

Market impact: This narrative drives interest in rare disease treatments, benefiting biotech companies like Every Cure. Increased awareness may boost demand for rare disease therapies, potentially improving valuations in the sector. It also highlights the need for innovative drug development strategies, as traditional methods are often cost-prohibitive.

What to watch next: In Q2 2023, monitor Every Cure's clinical trial updates for its repurposed drug candidates. Additionally, track regulatory decisions on rare disease therapies, such as the FDA's review of potential treatments for FOXG1 syndrome, to gauge the impact of increased awareness and advocacy on market access and approvals.