Williams Syndrome - affects an estimated 1 in 7,500 to 10,000 people
Smith-Magenis syndrome affects at least 1 in 25,000 individuals worldwide. However, researchers believe that many people with this condition are not diagnosed, so the true prevalence may be closer to 1 in 15,000 individuals.
Why go after all these rare diseases when AD results are around the corner (2022)?
"When a new use is discovered for a drug, it can earn an additional three years of protection under FDA rules. Suppose a drug designed to treat clinical depression was found to treat another disorder, like ADHD. In such cases, the drug can gain three additional years of patent protection under new use rules."