Gene Therapy in Patients With Mucopolysaccharidosis Disease

  • STATUS
    Recruiting
  • End date
    Sep 30, 2025
  • participants needed
    10
  • sponsor
    Fondazione Telethon
Updated on 8 December 2021

Summary

This study investigated the safety and efficacy of gene therapy approaches for Mucopolysaccharidosis type VI disease caused by the deficiency of arylsulfatase B (ARSB) enzyme. The aim of the study is to evaluate the safety and efficacy of the treatment.

Description

Mucopolysaccharidosis type VI disease is involved in Lysosomal Storage Disorder. The MPS VI disease is characterized by growth retardation, corneal clouding, cardiac valve disease, organomegaly, skeletal dysplasia, without central nervous system involvement.

Details
Condition Maroteaux-Lamy Syndrome
Treatment AAV2/8.TBG.hARSB
Clinical Study IdentifierNCT03173521
SponsorFondazione Telethon
Last Modified on8 December 2021

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