Mucopolysaccharidosis iii-A

Clinical trials for Mucopolysaccharidosis iii-A explained in plain language.

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Can a weekly infusion reach the brain to fight a rare childhood disease?

Disease control Ongoing

This trial tests an experimental drug called JR-441 in children with mucopolysaccharidosis type IIIA (MPS IIIA), a rare genetic condition that harms the brain and body. Researchers give the drug as a weekly intravenous infusion to see if it is safe and tolerable. They also measur…

Matched conditions: MUCOPOLYSACCHARIDOSIS III-A

Phase 1/2 • Sponsor: JCR Pharmaceuticals Co., Ltd. • Aim: Disease control

Last updated Sep 06, 2026 00:00 UTC

Mucopolysaccharidosis iii-A

Get alerted when new Mucopolysaccharidosis iii-A trials appear

Can a weekly infusion reach the brain to fight a rare childhood disease?