Disorder of peroxisomal transporter
MONDO:0100372Any peroxisomal single enzyme/protein defect that disrupts peroxisomal transport.
Also known as: disorder of peroxisomal transporter defect
42 clinical trials for this condition and its sub-types.
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Broader categories
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Halted gene therapy study raises questions for AMN patients
Disease control TerminatedThis early-stage trial tested a gene therapy called SBT101 for adrenomyeloneuropathy (AMN), a rare nerve disease that causes walking difficulties. Eight adults received either the therapy or a sham procedure. The study was terminated early, so we have limited data on safety and e…
Phase: PHASE1, PHASE2 • Sponsor: SwanBio Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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Rare disease mystery: scientists watch AMN progress in hopes of finding a cure
Knowledge-focused TerminatedThis study followed 65 adult men with a rare inherited nerve disease called AMN (a form of spastic paraplegia) to understand how their symptoms change over time. Researchers collected data on walking ability and quality of life. The goal was to fill gaps in knowledge about the di…
Sponsor: SwanBio Therapeutics, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:56 UTC