Rare

Donna and Ashley Appell in Hospital

“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 Hermansky-Pudlak 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.”

— Icarus Films

Film Accolades

• Best Feature Film, Brooklyn Girl Film Festival 2012
• Special Jury Award, Long Island Film Festival 2013
• Official Selection, 2014 Association of Clinical Research Professionals Conference
• Official Selection, 2013 DIA National Conference
• Official Selection, 2013 Arizona Science Center
• Official Selection, 2013 Bay Area Science Festival
• Official Selection, 2012 Seattle Science Festival
• Official Selection, 2012 Cannes Film Market
• Official Selection, 2012 National Institutes of Health Science Education Conference