What Precision Medicine Means for Rare Diseases
Join iBIO and PACT for a breakfast on March 8th in Springfield Illinois with a leading reasearchers participating in the Cohort Program of President Obama’s Precision Medicine Initiative (PMI).RSVP for the Breakfast
The PMI Cohort Program is a landmark longitudinal research effort that aims to engage 1 million or more U.S. participants to improve disease prevention and treatment measures based on individual differences in lifestyle, environment and genetics.
About Rare Diseases
Although individually any rare disease affects fewer than 200,000, as a collective, there are nearly 7,000 rare diseases afflict as many as 30 million people in the US, 15 million of which are children. Or another way to look at it, 1 in 10 Americans are suffering from rare diseases. Many rare diseases result in premature death of infants & young children or are fatal in early adulthood, 95% of rare diseases are without an FDA approved treatment or therapy. Even getting a diagnosis can be a struggle, patients are often misdiagnosed or undiagnosed.
Some 80% of an estimated 7,000 rare diseases involve a genetic component. Precision medicine takes advantage of these genetic components to tailor treatments to those patients most likely to respond. Rather than administering therapies based solely on the disease, new strategies to identify biomarkers help select the best therapies for individual patients.
A new report from the Personalized Medicine Coalition (PMC) shows that in 2016, for the third year in a row, personalized medicines accounted for more than 20 percent of the new molecular entities (NMEs) approved by the U.S. Food and Drug Administration (FDA). The FDA also approved several significant new personalized medicine indications for previously approved drugs in 2016. These approvals redefine the drugs’ intended populations and provide patients with effective personalized treatment options.
The iBIO/PACT breakfast event is part of the Illinois Rare Disease Day coordinated by NORD Illinois. The full day activities include:
8:30 am – 12:00 pm – Exhibits @ Capitol Rotunda
9:30 am – 10:15 am – Legislative Briefing @ Rm 402a
10:30 am – 11:00 am – Legislative Update @ Rm 402a
11:00 am – 2:00 pm – Legislative Visits
If you are interested in participating in Illinois Rare Disease Day, please contact NORD Illinois, Illinois.email@example.com