MUCOLIPIDOSIS TYPE IV
Clinical trials for MUCOLIPIDOSIS TYPE IV explained in plain language.
Never miss a new study
Get alerted when new MUCOLIPIDOSIS TYPE IV trials appear
Sign up with your email to follow new studies for MUCOLIPIDOSIS TYPE IV, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
Genom att skicka in godkänner du våra Användarvillkor
-
Experimental gene therapy hopes to tackle rare childhood disease
Disease control ENROLLING_BY_INVITATIONThis early-stage study tests a gene therapy called AAV9.hMCOLN1co in one child with Mucolipidosis Type IV, a rare genetic disorder. The goal is to see if the treatment is safe and can help control the disease. Participants must be between 1.5 and 8 years old and have a confirmed …
Matched conditions: MUCOLIPIDOSIS TYPE IV
Phase: PHASE1 • Sponsor: The Children's Hospital of Zhejiang University School of Medicine • Aim: Disease control
Last updated May 26, 2026 10:35 UTC
-
New study maps the journey of rare disease MLIV
Knowledge-focused OngoingThis study looks back at medical records of 50 people with mucolipidosis type IV (MLIV) to understand how the disease progresses. Researchers will track when children reach or lose developmental milestones, how movement and vision change, and what brain scans show. No new treatme…
Matched conditions: MUCOLIPIDOSIS TYPE IV
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated May 26, 2026 11:07 UTC