When you have a loved one with a rare disease, the importance of persevering to find new, innovative treatments takes on new meaning. This year on Rare Disease Day, I am excited to celebrate the advances we saw in treatments last year and the hope we have for continued progress in the years ahead.
Rare diseases are those that affect fewer than 200,000 people in the United States, but with more than 7,000 different rare diseases known today, nearly 1 in 10 Americans is living with a rare disease. Eighty percent of rare diseases – including the one impacting my family – are genetic in origin, making the rarity of each disease often unknown and treatment options especially limited. Today, approved treatments are available for just 5 percent of rare diseases.