Life For Liam
Fighting to Survive
Niemann Pick Type-C
Niemann Pick Type-C (NPC) is a fatal rare genetic disease that impacts every cell in the body. Often called "Childrens's Alzheimers", the disease progresses rapidly from birth, interrupting neurological development as the cells in the brain and nervous system die off. This incurable disease causes damage that is irreversible and is ultimately fatal. Most children diagnosed with NPC will not live to be 10 years old without treatment.
Due to the complexity of the disease and inconsistency of disease progresson, NPC kiddos often struggle for years before getting a confirmed diagnosis. A few experimental treatment have shown to help slow down the rate of progression of the disease if given early enough. With NPC every day counts.
We are extremely blessed to have learned Liam's diagnosis early and we are grateful there is a strong international community established around NPC.
Decades of effort has been put into research, development of medicines, and programs to help with early diagnosis.
Sadly, even with having a confirmed diagnosis at 2.5 months of age, our family fought for almost a year and a half to get Liam access to any of the experimental treatments. Liam was not sick enough to be allowed access to medicine that could prolong his life.
No child with a fatal genetic disorder should have to wait to recieve life extending treatments, nor should these children have to risk being put on a placebo as part of a clinical trial.
Help us get better treatment for Liam and help us push for change for our children!