Tag: rare disease research

Gene therapy companies are powering a revolution in rare disease cures by offering treatments that target the genetic cause of […]

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 […]