Mucopolysaccharidosis Type 1 (MPS I, Hurler Syndrome) Latest Advances

Lipid Nanoparticle-Delivered mRNA Therapy Corrects Neonatal Murine MPS I-H.

Journal: Human gene therapy

Published: March 26, 2026

Over ten years of newborn screening for LSDs in Tuscany (Italy): Epidemiology, novel variants, and the pseudodeficiency burden.

Journal: Molecular genetics and metabolism

Published: February 20, 2026

First-in-human intracisternal dosing of RGX-111 in severe MPS I is well-tolerated and generates sustained neurodevelopment without HSCT.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: February 10, 2026

Carpal tunnel syndrome in mucopolysaccharidosis type I: clinical, surgical and histopathological findings.

Journal: The Journal of hand surgery, European volume

Published: February 09, 2026

Carpal tunnel syndrome in mucopolysaccharidosis type I: clinical, surgical and histopathological findings.

Journal: The Journal of hand surgery, European volume

Published: February 09, 2026

Liposomal CRISPR/Cas9-Mediated Local Genome Editing for Joint Disease in Mucopolysaccharidosis Type I.

Journal: Pharmaceutics

Published: January 28, 2026

Phenotypes of Elosulfase Alfa-Induced Immediate Hypersensitivity Reactions and Long-term Outcomes of Desensitization in Mucopolysaccharidosis IVA.

Journal: International archives of allergy and immunology

Published: January 14, 2026

Beyond Detection: Comparing State-Based Newborn Screening Methods for Effective Mucopolysaccharidosis I Diagnosis.

Journal: International journal of neonatal screening

Published: January 07, 2026

Neurophysiological Characteristics of Upper Extremity Neuropathy in Three Young Patients with Mucopolysaccharidosis Type I and II in a Five-Year Observation-A Case Series Study.

Journal: Neurology international

Published: December 19, 2025

From trial to transformation: insights into hematopoietic stem cell-gene therapy for Hurler syndrome.

Journal: Expert opinion on biological therapy

Published: December 17, 2025

Case Report: Compound heterozygous mutations in the IDUA gene causing mucopolysaccharidosis type I with uterine developmental abnormality.

Journal: Frontiers in pediatrics

Published: December 15, 2025

Cerebrospinal fluid heparan sulfate as a biomarker for neuronopathic mucopolysaccharidoses: Rationale and regulatory challenges.

Journal: Molecular genetics and metabolism

Published: November 26, 2025

Mucopolysaccharidosis Type 1 (MPS I, Hurler Syndrome) Latest Advances

Find the Latest Research About Mucopolysaccharidosis Type 1 (MPS I, Hurler Syndrome)

Lipid Nanoparticle-Delivered mRNA Therapy Corrects Neonatal Murine MPS I-H.

Lipid Nanoparticle-Delivered mRNA Therapy Corrects Neonatal Murine MPS I-H.

Over ten years of newborn screening for LSDs in Tuscany (Italy): Epidemiology, novel variants, and the pseudodeficiency burden.

Over ten years of newborn screening for LSDs in Tuscany (Italy): Epidemiology, novel variants, and the pseudodeficiency burden.

First-in-human intracisternal dosing of RGX-111 in severe MPS I is well-tolerated and generates sustained neurodevelopment without HSCT.

First-in-human intracisternal dosing of RGX-111 in severe MPS I is well-tolerated and generates sustained neurodevelopment without HSCT.

Carpal tunnel syndrome in mucopolysaccharidosis type I: clinical, surgical and histopathological findings.

Carpal tunnel syndrome in mucopolysaccharidosis type I: clinical, surgical and histopathological findings.

Carpal tunnel syndrome in mucopolysaccharidosis type I: clinical, surgical and histopathological findings.

Carpal tunnel syndrome in mucopolysaccharidosis type I: clinical, surgical and histopathological findings.

Liposomal CRISPR/Cas9-Mediated Local Genome Editing for Joint Disease in Mucopolysaccharidosis Type I.

Liposomal CRISPR/Cas9-Mediated Local Genome Editing for Joint Disease in Mucopolysaccharidosis Type I.

Phenotypes of Elosulfase Alfa-Induced Immediate Hypersensitivity Reactions and Long-term Outcomes of Desensitization in Mucopolysaccharidosis IVA.

Phenotypes of Elosulfase Alfa-Induced Immediate Hypersensitivity Reactions and Long-term Outcomes of Desensitization in Mucopolysaccharidosis IVA.

Beyond Detection: Comparing State-Based Newborn Screening Methods for Effective Mucopolysaccharidosis I Diagnosis.

Beyond Detection: Comparing State-Based Newborn Screening Methods for Effective Mucopolysaccharidosis I Diagnosis.

Neurophysiological Characteristics of Upper Extremity Neuropathy in Three Young Patients with Mucopolysaccharidosis Type I and II in a Five-Year Observation-A Case Series Study.

Neurophysiological Characteristics of Upper Extremity Neuropathy in Three Young Patients with Mucopolysaccharidosis Type I and II in a Five-Year Observation-A Case Series Study.

From trial to transformation: insights into hematopoietic stem cell-gene therapy for Hurler syndrome.

From trial to transformation: insights into hematopoietic stem cell-gene therapy for Hurler syndrome.

Case Report: Compound heterozygous mutations in the IDUA gene causing mucopolysaccharidosis type I with uterine developmental abnormality.

Case Report: Compound heterozygous mutations in the IDUA gene causing mucopolysaccharidosis type I with uterine developmental abnormality.

Cerebrospinal fluid heparan sulfate as a biomarker for neuronopathic mucopolysaccharidoses: Rationale and regulatory challenges.

Cerebrospinal fluid heparan sulfate as a biomarker for neuronopathic mucopolysaccharidoses: Rationale and regulatory challenges.