HOPE FOR MARIAN

Children and adults are in the fight of their lives against fatal Niemann-Pick Type C (NPC). Without treatment and a cure they will lose their ability to move, speak, eat, breathe and live. This disease strikes all ages, most frequently children. It's the short term memory loss of Alzheimer's, the tremors of Parkinson's and the systemic decline of ALS. The disease is always fatal, first striking from infancy to adulthood with progressive memory loss, seizures, choking, and loss of speech and movement. There is no cure or FDA approved therapy, and access to limited experimental therapies are restrictive. We are working to change this.

We give a voice to patients around the world advocating universal access for life sustaining medicines for this generation of children and adults fighting NPC.