Imagine waiting for years to learn what is ailing your sick child, only to receive a diagnosis that you’ve never heard of. That’s often the plight of a parent of a child with a rare disease.
CHOC works to diagnose, treat and support patients and families with rare conditions. Here are some facts on rare diseases:
- In the United States, a condition is considered “rare” it affects fewer than 200,000 persons.
- If all people with rare diseases lived in one country, it would be the world’s third most populous country.
- Approximately 50 percent of patients affected by rare diseases are children and 30 percent of these children will not live to see their fifth birthday.
- 95 percent of rare diseases have no Food and Drug Administration-approved drug treatment.
- Many pediatric cancers make the list of rare diseases.
But despite these statistics, patients with rare diseases and their families are most certainly not alone – and there is hope.
In addition to finding support from CHOC, they can also lean on The Global Genes Project, an advocacy organization based in Aliso Viejo. The group helps parents and patients through their diagnostic odyssey and supports them through their rare disease treatment.
The best defense against the progression of rare disease is knowledge, support and medical facilities with a strong eye towards innovative technologies and treatments.
Learn more about The Global Gene Project, and find resources and more information about rare diseases.
CHOC Hospital was named one of the nation’s best children’s hospitals by U.S. News & World Report in its 2020-21 Best Children’s Hospitals rankings and ranked in the cancer specialty.