Category: Rare Disease

In years past, there were no viable therapies for rare diseases. Today, the barrier to viable therapies is the cost associated with their development.

There are 7,000 known rare diseases in the world. Each of them affects less than 1% of the global population, […]

Historically, developing rare disease cures and treatments has been challenging. The small, globally dispersed patient population made it difficult and […]

Approximately 7,000 rare diseases affect 350-500 million people worldwide. The majority of these (80%) are monogenic, so-called because these genetic […]

Of the 7,000 known rare diseases affecting 350 to 400 million people worldwide, most have a genetic basis and it […]

An emerging drug development model has the potential to reduce the cost of treatments for rare diseases. What is a […]