It is estimated that 400 million people suffer from a rare disease globally, with approximately 7,000 distinct types of rare and genetic diseases identified. On average, it takes eight years before rare disease patients receive an accurate diagnosis and even then, the majority of those diagnosed lack access to approved therapies for targeted treatment. Many of these rare diseases affect children, with irreversible debilitating and painful impact that is often fatal. Thus, it is essential to identify patients early and connect them to treatment as soon as possible to improve patient outcomes.
Beyond the single asset: The rise of combination and synergistic innovation in neurology drug development
Introduction Historically, drug development within the neurology space has been characterized by the search for individual therapies that can arrest complex...