News, Events & Blogs
Redefining the future of Rare Diseases: The Power of Gene Therapy
Approximately 7,000 rare diseases are known today, and about 80% of them have a genetic basis. For many, there are no approved treatments. Gene therapy provides a personalized approach by addressing the underlying mutation.
Economics and Politics in Rare Disease Treatments: The Rising Cost and Challenges
Orphan drug approvals are rising, offering hope for rare disease patients — but at an average cost of $370,000 per year. With policy shifts, Medicare loopholes, and falling research funding, the future of rare disease treatment hangs in the balance.