In an era of healthcare reform and heightened scrutiny of pharmaceutical innovation, America’s leadership in rare disease drug development stands out as a remarkable achievement.

By: Juan Francisco Cabello, MD and Marshall Summar, MD Latin America offers a dynamic and promising environment for rare disease research, characterized by growing momentum

By Rob Freishtat, MD, MPH, president and co-founder, Uncommon Cures, and Matthew Stares, senior vice president, global real estate, architecture and development, MillerKnoll More than

By Simon Frost and Marshall Summar Rare diseases, defined as conditions affecting fewer than 200,000 individuals in the United States, present significant challenges to the

Introduction Rare disease clinical trials are a vital intersection of scientific discovery and ethical responsibility. These trials provide hope for patients with life-altering conditions but

Rare disease research and drug development are at a transformative crossroads, with the FDA uniquely positioned to lead the charge in accelerating innovation. By embracing

Why Now is the Time to Invest in Rare Disease Therapies As a new administration takes charge and the spotlight intensifies on the challenges posed

We live in an era of extraordinary scientific advancement, and as a result, we are seeing rapid growth in the development of new drugs. When