RARE

RARE follows an extraordinary mother in a race against time to find a treatment for her daughters rare genetic disease When Donna Appell learned that her infant daughter Ashley had an extremely rare genetic disease that would kill her in thirty years she set out to track down every person in the world with HermanskyPudlak Syndrome HPS Realizing that no one was going to help cure just one child Donna forms an advocacy group and travels throughout the US and Puerto Rico to gather as many patients as possible who suffer from HPS which includes albinism blindness a bleeding disorder and often a fatal pulmonary fibrosis By the time Ashley turns twenty Donna under insurmountable odds has achieved something incredible the advocacy group she started is now in the hundreds and the NIH has agreed to start a clinical trial