Tag: Orphan drugs

While it's estimated that over 7,000 distinct rare diseases exist, with 95% lacking an FDA-approved treatment, the question "Are rare diseases treatable?" is increasingly answered with a resounding "Yes" for a growing number of conditions. Advancements in medical science have shifted the focus from merely managing symptoms to developing groundbreaking, targeted therapies.

With approximately 7,000 to 10,000 rare diseases identified, the number that have a truly curative treatment is surprisingly small. This poses a critical question for millions of patients and their families: **How many rare diseases have cures?**

According to the National Institutes of Health, over 25 million Americans are living with a rare disease, many of which have no cure. The daunting question, 'What is a rare disease with no cure?', opens a window into the complex world of these often-overlooked and devastating medical conditions.