Cassidy and Addison Hempel received an experimental drug that was injected straight to their spines in hopes of finding a cure for their rare and highly fatal disease. It is their mother who pushed for this experiment to become possible.

Home-maker Chris Hempel of Reno, Nevada persuaded scientists to contribute their time and research for her 7-year-old twins. She also found a way to get the government on her side and approve of her daughters’ extremely unusual experiment. Hempel told everyone that getting help for her kids and fighting off rare diseases ought not to be very difficult.

However, it is understandably challenging to ask drug companies to fund and take interest in a very expensive research for a disease or a disorder so rare that it only affects a couple hundred people per year. Hempel’s daughters have Niemann-Pick Type C.

At present, treatment is only available for about 200 out of 7,000 rare diseases. These diseases are very rare that less than 200,000 people have them. However, if one would look at it from a different perspective and add all those diseases up, an average of 20 million Americans are affected by these “rare” diseases.

That is why a campaign has started to push more funding and research on treatments for rare diseases. This fall, the National Institutes of Health is expected to open a center designed for speeding up genetic discoveries that may contribute on the growing number of rare diseases.

The International Rare Disease Research Consortium is also fighting to achieve treatment for at least 200 more rare diseases before 2020 ends.