This site is dedicated to those who practice Hopeful Science: for individuals who live the reality of their genetic condition every day but who refuse to be defined by it, for the clinicians who commit their careers to treating these conditions with intelligence and compassion, and for the scientists, who develop the insights into the diseases and their potential treatments and who understand that it is through their science that they create a future of hope.

Hopeful Science is a nonprofit organization dedicated to making lives better by fostering more communication and information exchange among patients, caregivers, and scientists in order to develop novel approaches to translational medicine for genetic diseases.

This is the age of Precision Medicine. This is where we hold our hope.