Genethon
Clinical trials sponsored by Genethon, explained in plain language.
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Gene therapy trial offers hope for boys with rare immune disease
Disease control TerminatedThis study tested a gene therapy for boys with X-linked chronic granulomatous disease (X-CGD), a rare disorder that leaves them unable to fight off certain infections. The treatment involved taking the patients' own blood stem cells, adding a working copy of the faulty gene, and …
Phase: PHASE1, PHASE2 • Sponsor: Genethon • Aim: Disease control
Last updated May 17, 2026 13:02 UTC
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Gene therapy breakthrough offers hope for boys with rare immune disease
Disease control TerminatedThis study tested a gene therapy for boys with X-linked chronic granulomatous disease (X-CGD), a rare disorder that leaves them unable to fight off certain infections. The treatment involved taking the patients' own blood stem cells, adding a corrected gene, and returning them to…
Phase: PHASE1, PHASE2 • Sponsor: Genethon • Aim: Disease control
Last updated May 13, 2026 16:01 UTC