About Us

The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy. We can do more with the science we already have and bring life-saving treatments to millions of people suffering from rare diseases.  Currently, there are fewer than 400 approved treatments for 7000 rare diseases affecting more than 30 million Americans. read more

Recent News


Foundation President Keynotes World Orphan Drug Congress

Dr. Kakkis spoke on the importance of advocacy on behalf of rare disease patients, and how crucial this advocacy has been to the legislative progress seen by the rare disease community in recent years. Dr. Kakkis also discussed how important the Orphan Product Extensions Now, Accelerating Cures and Treatments Act (OPEN ACT, S.1421) is to rare disease patients.Read More

From Energy and Commerce: Max Goes to Washington for #CuresNow

A familiar face stopped by the Capitol last week, taking the #Path2Cures from his home in New Jersey to the U.S. Senate to drop off some of his famous artwork to urge Senate support for #CuresNow. Max visited 83 Senate offices across two days, leaving his original artwork at each one.Read More

FDA Seeks Applicants for Patient Representative Program

The U.S. Food and Drug Administration (FDA) is seeking applicants for its FDA Patient Representative Program for several disease areas including, cardiovascular disease, sarcoma, thyroid and bladder cancer. Applicants can be patients or caregivers, who have direct experience with the disease. If selected, individuals will participate on FDA Advisory Committees and provide direct input to inform the Agency’s approval process for drugs, biologics, and medical devices.Read More

California Bill to Improve Newborn Screening Passes First Legislative Hurdle

On April 13th, the California Senate Health Committee heard and unanimously passed SB 1095, which would speed and improve the state’s newborn screening system by screening for a disease once it’s added to the Recommended Uniform Screening Panel (RUSP).Read More

Nature Biotechnology Publishes EveryLife Article on Need for Improved Pathways for Rare Disease Therapies at FDA

Dr. Emil Kakkis and Max Bronstein of the EveryLife Foundation authored an article in the April issue of Nature Biotechnology, calling on the FDA to provide clearer guidance to sponsors looking to develop rare disease therapies using biomarkers, which could substantially accelerate the pace of development for rare disease therapeutics.Read More