Today, February 28, is Rare Disease Day, an annual international event that aims to raise awareness about the thousands of under-the-radar diseases that significantly impact people's lives. My daughter is one of those people.
Demoralized and despondent, I was back in my doctor's office for the umpteenth time in three years. Dr. Schorling walked in looking unsuitably chipper holding a scrap of paper torn from a note pad. So far all diagnostic roads led nowhere.
February marks progress in overturning terrible odds. The landscape has changed. Drug companies more and more recognize that rare diseases have provided some of the most important medical breakthroughs on record. The therapies they're funding are finding new and more applications.
In terms of the science, it seems that "rare diseases" are more likely to be neglected than so-called "neglected tropical diseases." Perhaps in terms of NTDs, it's not the diseases but the people who have been neglected?