Elpida Therapeutics Spc
Clinical trials sponsored by Elpida Therapeutics Spc, explained in plain language.
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One-Time gene shot aims to stop rare nerve disease in kids
Disease control Recruiting nowThis early-phase trial tests a single injection of gene therapy (ELP-02) into the spinal fluid of 8 children and young adults with CMT4J, a rare genetic nerve disease that causes muscle weakness. The therapy delivers a working copy of the FIG4 gene to nerve cells, potentially slo…
Phase: PHASE1, PHASE2 • Sponsor: Elpida Therapeutics SPC • Aim: Disease control
Last updated Jul 10, 2026 00:00 UTC
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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. …
Phase: PHASE1, PHASE2 • Sponsor: Elpida Therapeutics SPC • Aim: Disease control
Last updated Jul 10, 2026 00:00 UTC
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Hope for kids with rare paralysis: gene therapy trial launches
Disease control Recruiting nowThis phase 3 trial tests a one-time gene therapy called MELPIDA for children with SPG50, a rare genetic disease that causes progressive paralysis and developmental delays. The study will give the treatment via a spinal injection to 24 children aged 4 months to 6 years and compare…
Phase: PHASE3 • Sponsor: Elpida Therapeutics SPC • Aim: Disease control
Last updated Jun 27, 2026 12:10 UTC