• 15 Retinopathy in patients with mucopolysaccharidosis
• A novel splicing variant in GALNS in mucopolysaccharidosis IVA and the necessity of re-evaluating primer sequences
• A pictorial review of the radiographic skeletal findings in Morquio syndrome (mucopolysaccharidosis type IV)
• Abnormal Fundus Autofluorescence in Eyes with Morquio Syndrome
• Activity of daily living in mucopolysaccharidosis IVA patients: Evaluation of therapeutic efficacy
• Addressing the need for patient-friendly medical communications: adaptation of the 2019 recommendations for the management of MPS VI and MPS IVA
• Adjunct diagnostic value of radiological findings in mucopolysaccharidosis type IVa-related thoracic spinal abnormalities: a pilot study
• Airway Abnormalities in Adult Mucopolysaccharidosis and Development of Salford Mucopolysaccharidosis Airway Score
• Airway management in mucopolysaccharidosis: a retrospective case series review
• Airway Management of the Deformed Trachea Using T-Tube Stents in Patients with Mucopolysaccharidosis Type IVA
• An adolescent case of sellar osteochondromyxoma in the setting of spondyloepiphyseal dysplasia
• An investigation of different intracellular parameters for Inborn Errors of Metabolism: Cellular stress, antioxidant response and autophagy
• Analysis of urinary oligosaccharide excretion patterns by UHPLC/HRAM mass spectrometry for screening of lysosomal storage disorders
• Anesthetic Concerns of Children With Skeletal Dysplasia
• Bone Growth Induction in Mucopolysaccharidosis IVA Mouse
• Cardiac involvement in MPS patients: incidence and response to therapy in an Italian multicentre study
• Causes of death in mucopolysaccharidoses
• Cervical Disorders in Mucopolysaccharidosis IVA-Morquio disease
• Changes in Corneal Clouding Over Time in Patients With Mucopolysaccharidosis
• Clinical characteristics of 111 cases with mucopolysaccharidosis A
• Clinical characteristics of 111 cases with mucopolysaccharidosis ⅣA
• Clinical features and health-related quality of life in adult patients with mucopolysaccharidosis IVA: the Spanish experience
• Clinical Utility of Elosulfase Alfa in the Treatment of Morquio A Syndrome
• Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice
• Comparison between Nanoparticle Encapsulation and Surface Loading for Lysosomal Enzyme Replacement Therapy
• Comparison of growth dynamics in different types of MPS: an attempt to explain the causes
• Comprehensive Preventive and Therapeutic Oral Health Care: A Case Report of Mucopolysaccharidosis Type IV A in a Pediatric Patient
• Consensus statement on enzyme replacement therapy for mucopolysaccharidosis IVA in Central and South-Eastern European countries
• Cranio-cervical decompression associated with non-instrumented occipito-C2 fusion in children with mucopolysaccharidoses: Report of twenty-one cases
• Delayed diagnosis of mild mucopolysaccharidosis type IVA
• Delayed Onset Post-Operative Neurologic Deficit in a Patient With Mucopolysaccharidosis type VI: A Case Report
• Delivery and assessment of a CRISPR/nCas9-based genome editing system on in vitro models of mucopolysaccharidoses IVA assisted by magnetite-based nanoparticles
• Demographic, clinical, and ancestry characterization of a large cluster of mucopolysaccharidosis IV A in the Brazilian Northeast region
• Dens Growth After Surgical Fixation in Children with Morquio-Brailsford Disease
• Dens growth following surgical fixation in children with Morquio-Brailsford disease
• Dental Considerations for the Treatment of Patients with Morquio Syndrome
• Differences in gene expression patterns, revealed by RNA-seq analysis, between various Sanfilippo and Morquio disease subtypes
• Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis
• Efficient CRISPR/Cas9 nickase-mediated genome editing in an in vitro model of mucopolysaccharidosis IVA
• Elosulfase alfa in the treatment of mucopolysaccharidosis type IVA: insights from the first managed access agreement
• Enhanced Efficiency of the Basal and Induced Apoptosis Process in Mucopolysaccharidosis IVA and IVB Human Fibroblasts
• Epidemiology of mucopolysaccharidoses (MPS) in United States: challenges and opportunities
• Evaluation of bone health in patients with mucopolysaccharidosis
• Evaluation of cardiac findings in mucopolysaccharidosis
• Evaluation of Gait Pattern and Lower Extremity Kinematics of Children with Morquio Syndrome (MPS IV)
• Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS)
• Evidence of epigenetic landscape shifts in mucopolysaccharidosis IIIB and IVA
• Exome Sequencing Identifies a Biallelic GALNS Variant (p.Asp233Asn) Causing Mucopolysaccharidosis Type IVA in a Pakistani Consanguineous Family
• Findings from the Morquio A Registry Study (MARS) after 6 years: Long-term outcomes of MPS IVA patients treated with elosulfase alfa
• Functional results after carpal tunnel release in mucopolysaccharidosis
• Generation of two iPSC lines from patient with Mucopolysaccharidosis IV B type and autosomal recessive non-syndromic hearing loss 12
• Genotype and Phenotype Characterization of Patients with Mucopolysaccharidosis IV-A in Chile
• Hearing Loss in Mucopolysaccharidosis
• How did intraoperative neuromonitorization prevent tetraplegia?
• Hyo-Mental Angle and Distance: An Important Adjunct in Airway Assessment of Adult Mucopolysaccharidosis
• IgG-cleavage protein allows therapeutic AAV gene delivery in passively immunized MPS IIIA mice
• Impact of the COVID-19 pandemic on the standard of care for patients with lysosomal storage diseases: A survey of healthcare professionals in the Fabry, Gaucher, and Hunter Outcome Survey registries
• Impaired ion homeostasis as a possible associate factor in mucopolysaccharidosis pathogenesis: transcriptomic, cellular and animal studies
• Inflammatory process and oxidative/nitrative stress: in vivo study in mucopolysaccharidosis type IV A patients under long-term enzyme replacement therapy
• Investigation of GALNS variants and genotype-phenotype correlations in a large cohort of patients with mucopolysaccharidosis type IVA
• Liver-directed gene therapy corrects neurologic disease in a murine model of mucopolysaccharidosis type I-Hurler
• Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II
• Mandibular condyle morphology among patients with mucopolysaccharidosis: An observational study of panoramic radiographs
• Molecular basis of mucopolysaccharidosis IVA (Morquio A syndrome): A review and classification of GALNS gene variants and reporting of 68 novel variants
• Molecular characterization of a large cohort of mucopolysaccharidosis patients: Iran Mucopolysaccharidosis RE-diagnosis study (IMPRESsion)
• Molecular characterization of mucopolysaccharidosis type IVA patients in the Andean region of Colombia
• Morquio A Syndrome: Identification of Differential Patterns of Molecular Pathway Interactions in Bone Lesions
• Morquio B disease: a case report
• Morquio-Brailsford Syndrome: Analysis of Bone Mass and Relevant Clinical Considerations
• Morquio-like dysostosis multiplex presenting with neuronopathic features is a distinct GLB1-related phenotype
• Mucopolysaccharidoses diagnosis in the era of enzyme replacement therapy in Egypt
• Mucopolysaccharidosis IVA: Current Disease Models and Drawbacks
• Mucopolysaccharidosis type IV: report of 5 cases of Morquio Syndrome
• Natural Evolution of Morquio A Syndrome Caused by Two Heterozygous Mutations of the <em>GALNS</em> Gene
• Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunter syndrome
• Objectively measuring anterior segment alterations in the eyes of mucopolysaccharidoses: Its utility in early diagnosis of glaucoma
• Pharmacological Chaperones for β-Galactosidase Related to G_M1 -Gangliosidosis and Morquio B: Recent Advances
• Pharmacological property, mechanism of action and clinical study results of Pabinafusp Alfa (Genetical Recombination) (IZCARGO() I.V. Infusion 10 mg) as the therapeutic for Mucopolysaccharidosis type-II (Hunter syndrome)
• Pharmacological property, mechanism of action and clinical study results of Pabinafusp Alfa (Genetical Recombination) (IZCARGO^® I.V. Infusion 10 mg) as the therapeutic for Mucopolysaccharidosis type-II (Hunter syndrome)
• Plasma Proteomic Analysis in Morquio A Disease
• Posterior Atlantoaxial Fusion With C1-2 Pedicle Screw Fixation for Atlantoaxial Dislocation in Pediatric Patients With Mucopolysaccharidosis IVA (Morquio a Syndrome): A Case Series
• Radiographic Findings of Mucopolysaccharidosis and Comparison with Bone Mineral Density: A Study from Southeastern Turkey
• Repetitive, non-invasive imaging of neurodegeneration, and prevention of it with gene replacement, in mice with Sanfilippo syndrome
• Safety, pharmacokinetics and CNS distribution of tralesinidase alfa administered via intracerebroventricular infusion to juvenile cynomolgus monkeys
• Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy
• Specific GAG ratios in the diagnosis of mucopolysaccharidoses
• Specific heterozygous variants in MGP lead to endoplasmic reticulum stress and cause spondyloepiphyseal dysplasia
• Spinal cord compression in patients with mucopolysaccharidosis
• Successful Elosulfase Alfa Desensitization Protocol in a Patient With Morquio A Syndrome
• The deleterious variants of N-acetylgalactosamine-6-sulfatase (GalN6S) enzyme trigger Morquio a syndrome by disrupting protein foldings
• The development of a broad-spectrum retaining β-exo-galactosidase activity-based probe
• The experiences and support needs of siblings of people with mucopolysaccharidosis
• The GALNS p.P77R variant is a probable Gujarati-Indian founder mutation causing Mucopolysaccharidosis IVA syndrome
• The landscape of Mucopolysaccharidosis in Southern and Eastern European countries: a survey from 19 specialistic centers
• The Outcome of Allogeneic Hematopoietic Stem Cell Transplantation From Different Donors in Recipients With Mucopolysaccharidosis
• The youngest pair of siblings with Mucopolysaccharidosis type IVA to receive enzyme replacement therapy to date: A case report
• Tracheal Narrowing and Its Impact on Anesthesia Care in Patients With Morquio A (Mucopolysaccharidosis Type IVA): An Observational Study
• Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy
• Understanding bias when estimating life expectancy from age at death: a simulation approach applied to Morquio syndrome A
• Untargeted LC-HRMS metabolomics reveals candidate biomarkers for mucopolysaccharidoses