An Open-Label, Single-Arm, Multicenter Study of Intracerebral Administration of Adeno-Associated Viral Vectors Serotype rh10 Carrying the Human N-sulfoglucosamine sulfohydrolase(SGSH) cDNA for the Treatment ofMucopolysaccharidosis Type IIIA

Project: Research project

Description

An Open-Label, Single-Arm, Multicenter Study of Intracerebral Administration of Adeno-Associated Viral Vectors Serotype rh10 Carrying the Human N-sulfoglucosamine sulfohydrolase (SGSH) cDNA for the Treatment of Mucopolysaccharidosis Type IIIA
StatusActive
Effective start/end date1/25/192/28/21

Funding

  • Lysogene

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Mucopolysaccharidosis III
Multicenter Studies
Complementary DNA
Serogroup
N-sulfoglucosamine sulfohydrolase