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.
A cure doesn't exist for Niemann Pick Type-C, yet! We are set on finding one and we are getting close!
Recent advancements in the field of genetics and gene therapy have tremendous potential to cure diseases like NPC at the root of the disease, the DNA. Genetic disorders can be cured by either editing the DNA, gene editing, to correct mutated genes or by delivering the missing mRNA caused by the mutated genes. We are researching the use of mRNA as a gene therapy to cure NPC and the data so far is very promising!
Help us develop a treatment for Liam and push for change for our children!
Life For Liam & Friends Foundation 501(c)(3) Pending