Clinical Trial Update: Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA [Phase 1, Phase 2]

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Summary

Phase 1/2 clinical trial evaluating a gene therapy using modified autologous hematopoietic stem cells for Mucopolysaccharidosis Type IIIA; sponsor is the Univer…

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Diseases / indications

• Mucopolysaccharidosis Type Iiia

Drugs mentioned

• Genetically modified autologous hematopoietic stem cell therapy (modified autologous hematopoietic stem cells)

Snippet

Sponsor: University of Manchester | Conditions: Mucopolysaccharidosis Type IIIA | Phase 1, Phase 2 | Status: ACTIVE_NOT_RECRUITING