Life 4 Liam
Racing to cure childhood Alzheimer’s.
Life 4 Liam & Friends is a registered nonprofit. We support families living with Niemann-Pick Type-C and fund a genetic path to stop it.
Cholesterol fills the cell. Then the cell dies.
Children with NPC cannot clear cholesterol from their lysosomes. The cell floods, then fails. In the brain, that is neurodegeneration. Often called childhood Alzheimer’s, NPC is rare, rapid, and still looking for a cure.
See how NPC works
This started with one boy, and a race against time.
Liam’s family built Life 4 Liam & Friends to connect NPC families, tell the truth about the disease, and fund research that could change the ending.
The science we funded is in print.
Dr. Mark Schultz’s lab published the first mRNA therapy for NPC1 in Molecular Therapy Nucleic Acids. Lipid-nanoparticle mRNA restored the missing protein in cells and in mouse liver. It is already out as a standalone article, and it will still be the featured cover of the September 2026 issue.
There is still no cure. There is finally more than hope.
Two FDA-approved medicines arrived in 2024. Gene therapy is the work we are funding.
FDA-approved Miplyffa (arimoclomol)
The first FDA-approved medicine for NPC, taken with miglustat to help fragile NPC1 protein keep working.
FDA-approved Aqneursa (levacetylleucine)
A stand-alone oral medicine that helps lysosomes and brain cells work better.
Used in NPC care Miglustat
An older substrate-reduction therapy that slows the buildup of certain lipids, and is now paired with Miplyffa.
High Stakes for Hope: a Casino Royale to Cure Niemann Pick Type-C
February 27, 2027, 6:00 – 11:00 PM PST · Via Tivoli
A Casino Royale evening for NPC — black tie, high stakes, and funds for a genetic path to a cure. Saturday, February 27, 2027.
Event detailsHear the family, and the people standing with them.
Interviews and news pieces live on the Watch page — LVMPD, the Las Vegas Sun, Channel 3, and KTNV.
Watch interviews