As a member of the Rare Disease Caucus, the Assembly Health Committee, and as Vice Chair of the Select Committee on Infectious Diseases in High Risk Communities, I am a proud co-author of ACR 162, which declares February 28 Rare Disease Day in California.
A rare disease is defined as affecting fewer than 200,000 people in the United States. Over 7,000 of these diseases have been identified, but the FDA has approved treatments for less than 500. So we have lots more work to do.
Patients are often misdiagnosed or under diagnosed as the average time for an accurate diagnosis of a rare disease is 8 years. Unfortunately, 50 percent of patients suffering from these diseases are children, and 30 percent will not see their 5th birthday.
Eighty percent of rare diseases are genetically based. New innovations in gene therapy and drug protocols that help people live longer lives are being discovered, tested and prescribed every day. I am excited to learn about these new gene therapy protocols which are being developed right here in California which is ground zero for life science innovation. In fact, the earlier a diagnosis is made, the better the outcome. Therefore medical professionals are looking to screen newborns for a host of rare disorders to start treatments before they leave the hospital giving them a great head-start to life!
NORD, the National Organization for Rare Diseases, is a major player in efforts to foster greater awareness of rare diseases. Initiatives include connecting patients and caregivers and to develop relationships with key decision makers.
By increasing public awareness and funding for medical research and removing barriers to access, we can move toward eradicating some of mankind’s most serious, life threatening diseases.
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Minority Floor Leader Marie Waldron, R-Escondido, represents the 75th Assembly District in the California Legislature, which includes the communities of Bonsall, Escondido, Fallbrook, Hidden Meadows, Pala, Palomar Mountain, Pauma Valley, Rainbow, San Marcos, Temecula, Valley Center and Vista.