News, Events & Blogs
NIH's All of Us Program Reaches a Historic Milestone for Precision Medicine
The NIH's All of Us Research Program has reached a historic milestone, becoming the world's largest integrated genomics and health database. Learn how this achievement could accelerate rare disease research, improve precision medicine, and bring new hope to patients and families worldwide.
Congress Reauthorizes Rare Pediatric Disease PRV Program: A Major Win for 30 million Americans
U.S. Congress delivered a major victory for the rare disease community with the five-year reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) Program alongside increased federal research funding and several patient-centered healthcare reforms.
Redefining the future of Rare Diseases: The Power of Gene Therapy
Approximately 7,000 rare diseases are known today, and about 80% of them have a genetic basis. For many, there are no approved treatments. Gene therapy provides a personalized approach by addressing the underlying mutation.
Navigating Clinical Trials: A Guide for Rare Disease Patients
A condition is defined as a rare disease if it affects fewer than 200,000 individuals. Approximately 7,000 conditions meet this definition. In 2016, the United States Food and Drug Administration awarded $23 million dollars towards a four year period that supported research for 21 different rare diseases. However, most rare diseases lack disease-modifying therapies or effective medications for several reasons…