Mucopolysaccharidosis Type 3 (MPS III, Sanfilippo Syndrome) Latest Advances

Find the Latest Research About Mucopolysaccharidosis Type 3 (MPS III, Sanfilippo Syndrome)

Last Updated: 09/26/2026

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Found 1004 publications

Characterizing enteric pathology in MPS IIIA mice suggests disease-specific vulnerability among lysosomal storage disorders.

Characterizing enteric pathology in MPS IIIA mice suggests disease-specific vulnerability among lysosomal storage disorders.

Journal: JCI insight
Published: July 23, 2026

Pathogenic Variants in HGSNAT associated with Autosomal Recessive Retinitis Pigmentosa without Overt Sanfilippo Syndrome.

Pathogenic Variants in HGSNAT associated with Autosomal Recessive Retinitis Pigmentosa without Overt Sanfilippo Syndrome.

Journal: Retinal cases & brief reports
Published: June 26, 2026

Mucopolysaccharidosis type IIIA and IIIC phenotypic progression: A case series.

Mucopolysaccharidosis type IIIA and IIIC phenotypic progression: A case series.

Journal: Molecular genetics and metabolism reports
Published: May 16, 2026

Personalized Drug Repurposing Screen Identifies Patient-Specific Therapeutic Candidates for Mucopolysaccharidosis Type IIIB.

Personalized Drug Repurposing Screen Identifies Patient-Specific Therapeutic Candidates for Mucopolysaccharidosis Type IIIB.

Journal: Journal of personalized medicine
Published: May 12, 2026

Growth Patterns in MPS IVA and MPS IIIA: A Longitudinal Single-Center Study.

Growth Patterns in MPS IVA and MPS IIIA: A Longitudinal Single-Center Study.

Journal: Journal of clinical medicine
Published: April 19, 2026

Mapping Sanfilippo Syndrome: A Multisystem Clinicopathological Autopsy.

Mapping Sanfilippo Syndrome: A Multisystem Clinicopathological Autopsy.

Journal: Diagnostics (Basel, Switzerland)
Published: March 19, 2026

Molecular Modeling of N-Acetylglucosamine Binding to the I154R Mutant of NAGLU: Pathogenic Insights into Sanfilippo Syndrome Type B.

Molecular Modeling of N-Acetylglucosamine Binding to the I154R Mutant of NAGLU: Pathogenic Insights into Sanfilippo Syndrome Type B.

Journal: International journal of molecular sciences
Published: March 16, 2026

Human iPSC-derived neural progenitor gene therapy improves outcomes in a neuropathic lysosomal disease.

Human iPSC-derived neural progenitor gene therapy improves outcomes in a neuropathic lysosomal disease.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: January 26, 2026

Human iPSC-derived neural progenitor gene therapy improves outcomes in a neuropathic lysosomal disease.

Human iPSC-derived neural progenitor gene therapy improves outcomes in a neuropathic lysosomal disease.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: January 26, 2026

Novel HGSNAT Variants Identified in the Oldest Siblings With MPS IIIC: Functional Characterization and Literature Review.

Novel HGSNAT Variants Identified in the Oldest Siblings With MPS IIIC: Functional Characterization and Literature Review.

Journal: American journal of medical genetics. Part A
Published: January 16, 2026

Retinal Phenotype in Mucopolysaccharidosis Type III.

Retinal Phenotype in Mucopolysaccharidosis Type III.

Journal: American journal of ophthalmology
Published: December 29, 2025

Evaluation of GlcNAc-Configured Glycomimetics as Pharmacological Chaperones of NAGLU for the Treatment of Mucopolysaccharidosis IIIB.

Evaluation of GlcNAc-Configured Glycomimetics as Pharmacological Chaperones of NAGLU for the Treatment of Mucopolysaccharidosis IIIB.

Journal: Biomolecules
Published: December 20, 2025
Showing 1-12 of 1,004

Last Updated: 09/26/2026