Article

Three-Month Observational Data for the MPS IIIB Sentinel Subject Following AAV9 Mediated Gene Therapy

2026-06-09

Abstract excerpt

<h4>Background</h4> Mucopolysaccharidosis type IIIB (MPS IIIB) is a devastating neurodegenerative lysosomal storage disorder caused by alpha-N-acetylglucosaminidase (NAGLU) deficiency. There is currently no approved therapy. We report the 3-month outcomes of a novel intracerebroventricular (ICV) gene therapy in a child with MPS IIIB. <h4>Methods</h4> In an open-label, single-center, investigator-initiated trial...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: b6978c94-bf42-53cc-8980-f17fc56a16bb
DOI: 10.64898/2026.06.01.26354386

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Three-Month Observational Data for the MPS IIIB Sentinel Subject Following AAV9 Mediated Gene TherapyDOI 10.64898/2026.06.01.26354386Read

Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System–Directed, AAV9-Mediated Iduronate Sulfatase Gene TransferSemantic proximity Read

Therapeutic Options for Mucopolysaccharidosis II (Hunter Disease).Semantic proximity Read

Multi-year enzyme expression in patients with mucopolysaccharidosis type VI after liver-directed gene therapySemantic proximity Read

Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder?Semantic proximity Read

Targeting Neurological Aspects of Mucopolysaccharidosis Type II: Enzyme Replacement Therapy and BeyondSemantic proximity Read

Enzyme Replacement Therapy in Mucopolysaccharidosis Type VII: A Three-year Clinical Outcome Study of the First Taiwanese CaseSemantic proximity Read

Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.Semantic proximity Read

Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy.Semantic proximity Read

Targeting the Root Cause of Mucopolysaccharidosis IIIA with a New scAAV9 Gene Replacement VectorSemantic proximity Read

A Review Of The Clinical Outcomes In Idursulfase-Treated And Untreated Filipino Patients With Mucopolysaccharidosis Type II: Data From The Local Lysosomal Storage Disease RegistrySemantic proximity Read

Broad Functional Correction of Molecular Impairments by Systemic Delivery of scAAVrh74-hSGSH Gene Delivery in MPS IIIA MiceSemantic proximity Read

Phase I and II clinical trials for the mucopolysaccharidosesSemantic proximity Read