IGT Recognizes Rare Disease Day

This Sunday, February 28th marks the 14th Rare Disease Day. Patients, advocates, providers, and others around the world recognize this important day by raising awareness of rare diseases and efforts to find cures and treatments. This year, we join this community in a call for action that gives people living with rare diseases equal opportunities to realize their full participation in family, work, and social life.


There are approximately 300 million people living with a rare disease around the world. Seventy-two percent of rare diseases are genetic in nature, and 70 percent of those genetic diseases begin in childhood. Gene therapies, which are designed to treat the root cause of diseases, are already making a tangible difference in the lives of people with rare diseases and many more gene therapies are in the pipeline offering hope for more patients and their families.


At IGT, we know that gene therapies cannot realize their full potential if we don’t work together to address important policy issues, including access barriers, FDA modernization, and newborn screening.


According to a study released this week by the EveryLife Foundation for Rare Diseases, the estimated cost of 379 rare diseases in the United States amounted to nearly $1 trillion in 2019. Gene therapies have the potential to alleviate the burden of rare diseases on the healthcare system.


To that end, IGT is working to encourage early screening, support research and development, and remove barriers to access so that rare disease patients can thrive.


IGT is proud to support the rare disease community and is hopeful for the future of gene therapies in treating rare diseases. Visit our Advisory Council page to see how our members participated in this year’s Rare Disease Day and learn more about their ongoing efforts to support the community.

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The Institute for Gene Therapies advocates for a modernized regulatory and reimbursement framework that encourages the development of transformative gene therapies and promotes patient access. 

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