The experience of rare disease can be overwhelming for both patients and their families. Diagnoses are often difficult, involving repeated visits to multiple specialists. Once a diagnosis is made, patients live with severe restrictions and life expectations, not to mention the tumult of having hopes raised and then dashed through successive treatments. Despite being collectively known as ‘rare diseases’, they effect over 300 million people worldwide.
Cure Rare Disease (CRD), a Boston-based nonprofit biotechnology company, was born of such an experience, one shared by millions of patients and families around the world. Rich Horgan, founder and Executive Director, and Blavatnik Life Science Entrepreneur-in-Residence at Harvard Business School, has witnessed three generations of his family affected by Duchenne muscular dystrophy, a fatal, neuromuscular disease. His younger brother currently suffers from the disease. Through his family’s experience, Rich realized that one of the most significant obstacles to effective treatment is the lack of collaboration among researchers and length of time it takes to develop a therapeutic. As anyone who is touched by a fatal, rare disease knows, time is of the essence.