(Atavist Magazine) – When a little girl in Iowa was diagnosed with a rare incurable disease, her parents and their community came together to help her. They wound up making medical history.

To patients with severe rare diseases, the drugs that target their conditions can mean life itself. They must take the medications daily, weekly, monthly, or whatever is required, year after year, forever. It’s often said that you can’t put a price on life, but drug companies do just that. In 2010, Soliris, the drug that Richard’s lab helped advance for the treatment of aHUS, set an industry record when it was priced at more than $400,000 for a year’s supply. Soliris has since been eclipsed. Lenmeldy, a one-time treatment for metachromatic leukodystrophy, an often fatal genetic disease affecting the brain and nervous system, costs an estimated $4.2 million. The breakthroughs in C3G research introduced two more examples of sky-high drug prices: Fabhalta, an oral medication taken twice daily, costs nearly $600,000 for a year’s supply. Empaveli, which C3G patients receive through twice-weekly injections, clocks in at more than $500,000. (Read More)