Mucolipidosis type IV
MONDO:0009653A lysosomal disease characterized by psychomotor delay, progressive visual impairment, and achlorhydria.
Also known as: ML 4, ML IV, ML4, MLIV, Mucolipidosis IV, mucolipidosis IV, mucolipidosis type 4, mucolipidosis type IV
3 clinical trials for this condition and its sub-types.
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First gene therapy trial launches for rare childhood disease MLIV
Disease control By invitation onlyThis early-stage trial tests a gene therapy called AAV9.hMCOLN1co in one child with Mucolipidosis Type IV (MLIV), a rare genetic disorder. The therapy is given as a single injection into the spinal fluid. The main goal is to check safety, but researchers will also look for any si…
Phase 1 • Sponsor: The Children's Hospital of Zhejiang University School of Medicine • Aim: Disease control
Last updated Jun 27, 2026 09:03 UTC
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New study tracks rare disease MLIV to map its natural course
Knowledge-focused OngoingThis study looks back at medical records of 50 people with Mucolipidosis Type IV (MLIV) to learn how the disease typically progresses. Researchers want to know when children reach or lose developmental milestones, how their movement and vision change, and what lab results look li…
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:23 UTC