SPG50
Clinical trials for SPG50 explained in plain language.
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Gene therapy aims to halt rare childhood paralysis disorder
Disease control Recruiting nowThis trial tests a single injection of MELPIDA, a gene therapy that delivers a working copy of the AP4M1 gene directly into the spinal fluid of children with SPG50. SPG50 is a rare genetic condition that causes muscle stiffness, intellectual disability, and developmental delays. …
Matched conditions: SPG50
Phase: PHASE1, PHASE2 • Sponsor: Elpida Therapeutics SPC • Aim: Disease control
Last updated Jul 10, 2026 00:00 UTC
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Scientists launch major effort to track rare nerve disease in children
Knowledge-focused Recruiting nowThis study collects health information and biological samples from up to 700 people under 30 with early-onset hereditary spastic paraplegia (HSP). Researchers aim to better understand how the disease progresses over time and create a registry for future studies. Participants prov…
Matched conditions: SPG50
Sponsor: Boston Children's Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:02 UTC