Life 4 Liam
What exists today, and what we are still building.
Each card is the same shape: what it is, how it works, who it is for, and what it does not do. None of this replaces a conversation with an NPC clinician. Companies behind these medicines, and the family organizations walking with us, are listed on Community.
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.
Investigational Cyclodextrin
A molecular sponge being studied for its ability to pull trapped cholesterol out of the lysosome.
In research mRNA gene therapy
The Life 4 Liam approach: teach the cell to make the missing NPC1 protein using messenger RNA.
Essential care Supportive care
Therapy, nutrition, seizure care, school support, and daily help are part of treatment — not extras.
Research AAV gene therapy
Researchers can pack a working NPC1 gene into an engineered adeno-associated virus (AAV) capsid. That is gene addition — a different tool from the mRNA and prime-editing path Life 4 Liam funds.
Educational only. Not medical advice.