Gene Therapy in Patients With Mucopolysaccharidosis Disease

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


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.


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.

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

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