FAMILIAL CHYLOMICRONEMIA SYNDROME
Clinical trials for FAMILIAL CHYLOMICRONEMIA SYNDROME explained in plain language.
Never miss a new study
Get alerted when new FAMILIAL CHYLOMICRONEMIA SYNDROME trials appear
Sign up with your email to follow new studies for FAMILIAL CHYLOMICRONEMIA SYNDROME, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
Can a new shot tame a rare fat disorder in kids?
Disease control Recruiting nowThis trial tests whether olezarsen, an experimental drug given as a weekly injection, can lower dangerously high blood fat levels in children with familial chylomicronemia syndrome (FCS). FCS is a rare genetic condition that prevents the body from breaking down fats, leading to s…
Matched conditions: FAMILIAL CHYLOMICRONEMIA SYNDROME
Phase: PHASE3 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jul 31, 2026 00:00 UTC
-
Gene-Editing shot aims to tame deadly fat levels in kids
Disease control Recruiting nowThis early-stage trial tests a new gene-editing treatment called CS-121 in 15 children and teens (ages 4-18) with a rare condition causing extremely high triglycerides. The therapy uses lipid nanoparticles to deliver a base editor that targets the APOC3 gene in the liver. The mai…
Matched conditions: FAMILIAL CHYLOMICRONEMIA SYNDROME
Phase: EARLY_PHASE1 • Sponsor: Shanghai Jiao Tong University School of Medicine • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC