IJMS | Free Full-Text | Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art
Cureus | Hunter Syndrome: The Phenotype of a Rare Storage Disease | Article
Sanfilippo syndrome: consensus guidelines for clinical care | Orphanet Journal of Rare Diseases | Full Text
Outcomes of a Physician Survey on the Type, Progression, Assessment, and Treatment of Neurological Disease in Mucopolysaccharidoses - Maurizio Scarpa, Paul R. Harmatz, Bianca Meesen, Roberto Giugliani, 2018
Joint range of motion measurements in patients with MPS II (n=124). | Download Table
Diagnostics | Free Full-Text | Mucopolysaccharidosis: What Pediatric Rheumatologists and Orthopedics Need to Know
Impact of the Timing of Enzyme Replacement Therapy Initiation and Cognitive Impairment Status on Outcomes for Patients with Mucopolysaccharidosis II (MPS II) in the United States: A Retrospective Chart Review | Published
A molecular genetics view on Mucopolysaccharidosis Type II - ScienceDirect
The mucopolysaccharidoses and mucolipidosis II and III. | Download Table
PDF) Sanfilippo syndrome: consensus guidelines for clinical care
Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future | Journal of Human Genetics
IJMS | Free Full-Text | Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder?
Analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis II | Orphanet Journal of Rare Diseases | Full Text
Can serial cerebral MRIs predict the neuronopathic phenotype of MPS II? - Vollebregt - 2021 - Journal of Inherited Metabolic Disease - Wiley Online Library
Diagnostics | Free Full-Text | Diagnosis of Mucopolysaccharidoses
Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease – topic of research paper in Clinical medicine. Download scholarly article PDF and read for free on
IJMS | Free Full-Text | Mucopolysaccharidosis Type II: One Hundred Years of Research, Diagnosis, and Treatment
Analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis II | Orphanet Journal of Rare Diseases | Full Text
Impact of the Timing of Enzyme Replacement Therapy Initiation and Cognitive Impairment Status on Outcomes for Patients with Mucopolysaccharidosis II (MPS II) in the United States: A Retrospective Chart Review | Published
Clinical characteristics and genotypes of 201 patients with mucopolysaccharidosis type II in China: A retrospective, observational study - Zhong - Clinical Genetics - Wiley Online Library
Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease | Orphanet Journal of Rare Diseases | Full Text
Frontiers | Neurological, neurobehavioral, and radiological alterations in patients with mucopolysaccharidosis III (Sanfilippo's syndrome) in Brazil
Diagnostic algorithm for Hunter syndrome. From (Scarpa et al., 2011,... | Download Scientific Diagram
Professor Maurizio Scarpa: a metabolic paediatrician's experience with MPS II - YouTube
Biomolecules | Free Full-Text | Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement
