Time Is Short… As there is no proven treatment or cure for this cruel childhood disease!
From the age of five, it was apparent that the Marella’s daughter Dana, a happy, caring and energetic child, had visual and learning difficulties. After years of searching for answers to her challenges, Dana was diagnosed in 2002 at the age of eight with Niemann-Pick type C disease. In 2004, at the age of 5, Dana’s brother Andrew, the Marella’s 4th child, was also diagnosed with NPC. Dana’s visual and cognitive abilities, and her balance become progressively worse. On July 12th, 2013 Dana died from complications resulting from Niemann-Pick type C disease. Andrew is in the Phase 1 clinical trial of Cyclodextrin and with the benefit of other treatments we have helped find, Andrew continues to do well, though he is battling seizures.
Niemann-Pick Type C disease (NPC) is a genetic metabolic disorder in which harmful quantities of cholesterol and other fatty substances accumulate in the cells of the body such as the spleen, liver, lungs, bone marrow and, most dramatically, the brain.
Watch young Julia Kain’s faithful journey battling NP-C in our Gladys Knight Gala video presentation.
Learn about the unique NPC drug development collaborative, Support of Accelerated Research for NPC or SOAR-NPC, and the Cyclodextrin clinical trial at the National Institutes of Health!
Reflections on NPC Video – See the effects of NPC and learn about the benefits of NPC Research.
Andrea Marella of DART on the Today Show