- Home
- Publications
- Publication Search
- Publication Details
Title
The natural history of neurocognition in MPS disorders: A review
Authors
Keywords
Neurocognition, Natural history, Mucopolysaccharidoses
Journal
MOLECULAR GENETICS AND METABOLISM
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2021-03-12
DOI
10.1016/j.ymgme.2021.03.002
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII
- (2020) Raymond Y. Wang et al. MOLECULAR GENETICS AND METABOLISM
- Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI): Symptoms and the impact on function and activities of daily living (ADL)
- (2020) Dawn Phillips et al. MOLECULAR GENETICS AND METABOLISM
- Evidence of problems with “processing efficiency” in attenuated mucopolysaccharidosis type I
- (2020) Julie B. Eisengart et al. MOLECULAR GENETICS AND METABOLISM
- Vestronidase alfa: Recombinant human β-glucuronidase as an enzyme replacement therapy for MPS VII
- (2020) Jaclyn Cadaoas et al. MOLECULAR GENETICS AND METABOLISM
- Mucopolysaccharidosis Type I: A Review of the Natural History and Molecular Pathology
- (2020) Christiane S. Hampe et al. Cells
- Hematopoietic stem cell transplantation in mucopolysaccharidosis type IIIA: A case description and comparison with a genotype-matched control group
- (2020) Anja F. Köhn et al. Molecular Genetics and Metabolism Reports
- Therapy development for the mucopolysaccharidoses: Updated consensus recommendations for neuropsychological endpoints
- (2020) Johanna H. van der Lee et al. MOLECULAR GENETICS AND METABOLISM
- Natural history of cognitive development in neuronopathic mucopolysaccharidosis type II (Hunter syndrome): Contribution of genotype to cognitive developmental course
- (2020) Joo-Hyun Seo et al. Molecular Genetics and Metabolism Reports
- Design, baseline characteristics, and 18-24 months follow-up from the MPS IIIA natural history study
- (2019) Frits Wijburg et al. MOLECULAR GENETICS AND METABOLISM
- Natural history data for young subjects with Sanfilippo syndrome type B (MPS IIIB)
- (2019) Martha Solano Villarreal et al. MOLECULAR GENETICS AND METABOLISM
- Evidence of attention problems in Morquio syndrome
- (2019) Katherine Spurlock et al. MOLECULAR GENETICS AND METABOLISM
- Vestronidase Alfa: A Review in Mucopolysaccharidosis VII
- (2019) Emma H. McCafferty et al. BIODRUGS
- Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes
- (2019) Julie B. Eisengart et al. GENETICS IN MEDICINE
- Attention and corpus callosum volumes in individuals with mucopolysaccharidosis type I
- (2019) Kelly E. King et al. NEUROLOGY
- Therapeutic Options for Mucopolysaccharidoses: Current and Emerging Treatments
- (2019) Kazuki Sawamoto et al. DRUGS
- Developing interactions with industry in rare diseases: lessons learned and continuing challenges
- (2019) Susan A. Berry et al. GENETICS IN MEDICINE
- Ten years of enzyme replacement therapy in paediatric onset mucopolysaccharidosis II in England
- (2019) A. Broomfield et al. MOLECULAR GENETICS AND METABOLISM
- Analysis of the caregiver burden associated with Sanfilippo syndrome type B: panel recommendations based on qualitative and quantitative data
- (2019) Elsa Shapiro et al. Orphanet Journal of Rare Diseases
- Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study
- (2019) Agnes H. Chen et al. MOLECULAR GENETICS AND METABOLISM
- Pathway to diagnosis and burden of illness in mucopolysaccharidosis type VII – a European caregiver survey
- (2019) Alexandra Morrison et al. Orphanet Journal of Rare Diseases
- The attenuated end of the phenotypic spectrum in MPS III: from late-onset stable cognitive impairment to a non-neuronopathic phenotype
- (2019) Stephanie C. M. Nijmeijer et al. Orphanet Journal of Rare Diseases
- Markers of cognitive function in individuals with metabolic disease: Morquio syndrome and tyrosinemia type III
- (2018) James Blundell et al. COGNITIVE NEUROPSYCHOLOGY
- Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison
- (2018) Julie B Eisengart et al. GENETICS IN MEDICINE
- Observational Prospective Natural History of Patients with Sanfilippo Syndrome Type B
- (2018) Chester B. Whitley et al. JOURNAL OF PEDIATRICS
- ICV-administered BMN 250 (NAGLU-IGF2) is well tolerated and reduces heparan sulfate accumulation in the CNS of subjects with Sanfilippo syndrome type B (MPS IIIB)
- (2018) Nicole Muschol et al. MOLECULAR GENETICS AND METABOLISM
- Observing the advanced disease course in mucopolysaccharidosis, type IIIA; a case series
- (2018) Elsa Shapiro et al. MOLECULAR GENETICS AND METABOLISM
- Neurophysiology of hearing in patients with mucopolysaccharidosis type IV
- (2018) Kyoko Nagao et al. MOLECULAR GENETICS AND METABOLISM
- A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease
- (2018) Paul Harmatz et al. MOLECULAR GENETICS AND METABOLISM
- Epidemiology of Sanfilippo syndrome: results of a systematic literature review
- (2018) Tamás Zelei et al. Orphanet Journal of Rare Diseases
- Beneath the floor: re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome
- (2018) Elsa G. Shapiro et al. Orphanet Journal of Rare Diseases
- Mucopolysaccharidoses: overview of neuroimaging manifestations
- (2018) Manal Nicolas-Jilwan et al. PEDIATRIC RADIOLOGY
- Mucopolysaccharidosis III in Taiwan: Natural history, clinical and molecular characteristics of 28 patients diagnosed during a 21-year period
- (2018) Hsiang-Yu Lin et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART A
- Mucopolysaccharidosis type VI (MPS VI) and molecular analysis: Review and classification of published variants in the ARSB gene
- (2018) Rosella Tomanin et al. HUMAN MUTATION
- Haematopoietic stem cell transplantation for mucopolysaccharidosis type VII: A case report
- (2018) Luisa Sisinni et al. PEDIATRIC TRANSPLANTATION
- Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials
- (2018) Yulan Qi et al. CLINICAL PHARMACOKINETICS
- Intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A: A phase IIb randomized trial
- (2018) Frits A. Wijburg et al. MOLECULAR GENETICS AND METABOLISM
- Mucopolysaccharidosis VI pathophysiology diagnosis and treatment
- (2017) Paul Harmatz Frontiers in Bioscience-Landmark
- Clinical features of Mexican patients with Mucopolysaccharidosis type I
- (2017) A. Alonzo-Rojo et al. GENETICS AND MOLECULAR RESEARCH
- Quantitative clinical characteristics of 53 patients with MPS VII: a cross-sectional analysis
- (2017) Matthias Zielonka et al. GENETICS IN MEDICINE
- Mortality after hematopoietic stem cell transplantation for severe mucopolysaccharidosis type I: the 30-year University of Minnesota experience
- (2017) Nathan J. Rodgers et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Survival in idursulfase-treated and untreated patients with mucopolysaccharidosis type II: data from the Hunter Outcome Survey (HOS)
- (2017) Barbara K. Burton et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Mucopolysaccharidosis Type I Newborn Screening: Best Practices for Diagnosis and Management
- (2017) Lorne A. Clarke et al. JOURNAL OF PEDIATRICS
- Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial
- (2017) Marc Tardieu et al. LANCET NEUROLOGY
- Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure
- (2017) Johanna H. van der Lee et al. MOLECULAR GENETICS AND METABOLISM
- Cognitive and adaptive measurement endpoints for clinical trials in mucopolysaccharidoses types I, II, and III: A review of the literature
- (2017) Darren Janzen et al. MOLECULAR GENETICS AND METABOLISM
- Treatment of brain disease in the mucopolysaccharidoses
- (2017) Maurizio Scarpa et al. MOLECULAR GENETICS AND METABOLISM
- Quantitative neuroimaging in mucopolysaccharidoses clinical trials
- (2017) Igor Nestrasil et al. MOLECULAR GENETICS AND METABOLISM
- Assessments of neurocognitive and behavioral function in the mucopolysaccharidoses
- (2017) Elsa G. Shapiro et al. MOLECULAR GENETICS AND METABOLISM
- Minimal clinically important difference for the 6-min walk test: literature review and application to Morquio A syndrome
- (2017) Rudolf Schrover et al. Orphanet Journal of Rare Diseases
- Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry
- (2017) Joseph Muenzer et al. Orphanet Journal of Rare Diseases
- Efficacy and safety of intravenous laronidase for mucopolysaccharidosis type I: A systematic review and meta-analysis
- (2017) Alícia Dorneles Dornelles et al. PLoS One
- 12 year follow up of enzyme-replacement therapy in two siblings with attenuated mucopolysaccharidosis I: the important role of early treatment
- (2016) Orazio Gabrielli et al. BMC Medical Genetics
- Clinical course of sly syndrome (mucopolysaccharidosis type VII)
- (2016) Adriana M Montaño et al. JOURNAL OF MEDICAL GENETICS
- A Prospective Natural History Study of Mucopolysaccharidosis Type IIIA
- (2016) Elsa G. Shapiro et al. JOURNAL OF PEDIATRICS
- Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10year combined experience of 2 centres
- (2016) Arunabha Ghosh et al. MOLECULAR GENETICS AND METABOLISM
- Hematopoietic stem cell transplantation for Morquio A syndrome
- (2016) Hiromasa Yabe et al. MOLECULAR GENETICS AND METABOLISM
- Molecular diagnosis of 65 families with mucopolysaccharidosis type II (Hunter syndrome) characterized by 16 novel mutations in the IDS gene: Genetic, pathological, and structural studies on iduronate-2-sulfatase
- (2016) Motomichi Kosuga et al. MOLECULAR GENETICS AND METABOLISM
- Attenuated mucopolysaccharidosis II; Parental beliefs about the impact of disease on the quality of life of their children
- (2016) Archana Soni-Jaiswal et al. MOLECULAR GENETICS AND METABOLISM
- A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design
- (2016) K.V. Truxal et al. MOLECULAR GENETICS AND METABOLISM
- Activity of daily living for Morquio A syndrome
- (2016) Eriko Yasuda et al. MOLECULAR GENETICS AND METABOLISM
- Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study
- (2015) M. Aldenhoven et al. BLOOD
- Long-term cognitive follow-up in children treated for Maroteaux-Lamy syndrome
- (2015) Berendine J. Ebbink et al. JOURNAL OF INHERITED METABOLIC DISEASE
- First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient
- (2015) Joyce E. Fox et al. MOLECULAR GENETICS AND METABOLISM
- Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment
- (2015) Elsa G. Shapiro et al. MOLECULAR GENETICS AND METABOLISM
- Cognitive, medical, and neuroimaging characteristics of attenuated mucopolysaccharidosis type II
- (2015) Brianna Yund et al. MOLECULAR GENETICS AND METABOLISM
- Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships
- (2015) Nouriya A. Al-Sannaa et al. Orphanet Journal of Rare Diseases
- Mucopolysaccharidosis type IVA: Evidence of primary and secondary central nervous system involvement
- (2014) Felippe Borlot et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART A
- Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)-10-year follow-up of patients who previously participated in an MPS VI survey study
- (2014) Roberto Giugliani et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART A
- Clinical manifestations of 17 patients affected with mucopolysaccharidosis type VI and eight novelARSBmutations
- (2014) Piranit Nik Kantaputra et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART A
- Early treatment is associated with improved cognition in Hurler syndrome
- (2014) Michele D. Poe et al. ANNALS OF NEUROLOGY
- Histoire naturelle de la mucopolysaccharidose IV de type A (maladie de Morquio A)
- (2014) G. Baujat et al. ARCHIVES DE PEDIATRIE
- Enzyme replacement therapy for Mucopolysaccharidosis Type I among patients followed within the MPS Brazil Network
- (2014) Alícia Dorneles Dornelles et al. GENETICS AND MOLECULAR BIOLOGY
- The natural history of MPS I: global perspectives from the MPS I Registry
- (2014) Michael Beck et al. GENETICS IN MEDICINE
- Pain: a prevalent feature in patients with mucopolysaccharidosis. Results of a cross-sectional national survey
- (2014) Marion M. G. Brands et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Long-term experience with enzyme replacement therapy (ERT) in MPS II patients with a severe phenotype: an international case series
- (2014) Christina Lampe et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Burden of disease in patients with Morquio A syndrome: results from an international patient-reported outcomes survey
- (2014) Christian J Hendriksz et al. Orphanet Journal of Rare Diseases
- Clinical efficacy of Enzyme Replacement Therapy in paediatric Hunter patients, an independent study of 3.5 years
- (2014) Rosella Tomanin et al. Orphanet Journal of Rare Diseases
- New insights in mucopolysaccharidosis type VI: Neurological perspective
- (2013) Felippe Borlot et al. BRAIN & DEVELOPMENT
- Mucopolysaccharidosis type IIIB mutations in Chinese patients: Identification of two novel NAGLU mutations and analysis of two cases involving prenatal diagnosis
- (2013) Jia Tang et al. CLINICA CHIMICA ACTA
- MPS II: Adaptive Behavior of Patients and Impact on the Family System
- (2013) Mary Needham et al. Journal of Genetic Counseling
- Natural history of Sanfilippo syndrome type A
- (2013) Dakota Buhrman et al. JOURNAL OF INHERITED METABOLIC DISEASE
- The Morquio A Clinical Assessment Program: Baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects
- (2013) Paul Harmatz et al. MOLECULAR GENETICS AND METABOLISM
- Effects of idursulfase enzyme replacement therapy for Mucopolysaccharidosis type II when started in early infancy: Comparison in two siblings
- (2013) Go Tajima et al. MOLECULAR GENETICS AND METABOLISM
- Neurocognitive and neuropsychiatric phenotypes associated with the mutation L238Q of the α-L-iduronidase gene in Hurler–Scheie syndrome
- (2013) Alia Ahmed et al. MOLECULAR GENETICS AND METABOLISM
- Natural history of Sanfilippo syndrome in Spain
- (2013) Verónica Delgadillo et al. Orphanet Journal of Rare Diseases
- Brain and Spinal MR Imaging Findings in Mucopolysaccharidoses: A Review
- (2012) D.I. Zafeiriou et al. AMERICAN JOURNAL OF NEURORADIOLOGY
- Intellectual and neurological functioning in Morquio syndrome (MPS IVa)
- (2012) J. E. Davison et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Brain magnetic resonance imaging findings in patients with mucopolysaccharidosis VI
- (2012) Ana C. M. Azevedo et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Enzyme Replacement is Associated with Better Cognitive Outcomes after Transplant in Hurler Syndrome
- (2012) Julie B. Eisengart et al. JOURNAL OF PEDIATRICS
- An exploratory study of brain function and structure in mucopolysaccharidosis type I: Long term observations following hematopoietic cell transplantation (HCT)
- (2012) Elsa Shapiro et al. MOLECULAR GENETICS AND METABOLISM
- Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: A nationwide survey in Japan
- (2012) Akemi Tanaka et al. MOLECULAR GENETICS AND METABOLISM
- Enzyme Replacement Therapy in an Attenuated Case of Mucopolysaccharidosis Type I (Scheie Syndrome): A 6.5-Year Detailed Follow-Up
- (2012) Agnieszka Jurecka et al. PEDIATRIC NEUROLOGY
- Restricted joint range of motion in patients with MPS II: correlation with height, age and functional status
- (2011) Jolanta Marucha et al. ACTA PAEDIATRICA
- Enzyme replacement therapy for mucopolysaccharidosis II from 3 months of age: a 3-year follow-up
- (2011) A Tylki- Szymanska et al. ACTA PAEDIATRICA
- Genistein in Sanfilippo disease: A randomized controlled crossover trial
- (2011) Jessica de Ruijter et al. ANNALS OF NEUROLOGY
- Design, baseline characteristics, and early findings of the MPS VI (mucopolysaccharidosis VI) Clinical Surveillance Program (CSP)
- (2011) Christian J. Hendriksz et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Co-morbidity of Sanfilippo Syndrome type C and d-2-hydroxyglutaric aciduria
- (2011) M. Ali Pervaiz et al. JOURNAL OF NEUROLOGY
- Early Clinical Markers of Central Nervous System Involvement in Mucopolysaccharidosis Type II
- (2011) Joshua Holt et al. JOURNAL OF PEDIATRICS
- Hallazgos neurorradiológicos en una serie de pacientes con mucopolisacaridosis
- (2011) M.L. Calleja Gero et al. NEUROLOGIA
- Cognitive development in patients with Mucopolysaccharidosis type III (Sanfilippo syndrome)
- (2011) Marlies J Valstar et al. Orphanet Journal of Rare Diseases
- Natural Progression of Neurological Disease in Mucopolysaccharidosis Type II
- (2011) J. B. Holt et al. PEDIATRICS
- Mucopolysaccharidosis type III (Sanfilippo disease) in Sweden: clinical presentation of 22 children diagnosed during a 30-year period
- (2010) G Malm et al. ACTA PAEDIATRICA
- Incidence and natural history of mucopolysaccharidosis type III in France and comparison with United Kingdom and Greece
- (2010) Bénédicte Héron et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART A
- Correlation of Automated Volumetric Analysis of Brain MR Imaging with Cognitive Impairment in a Natural History Study of Mucopolysaccharidosis II
- (2010) Z. Fan et al. AMERICAN JOURNAL OF NEURORADIOLOGY
- Mucopolysaccharidosis type IIIC (Sanfilippo): early clinical presentation in a large Turkish pedigree
- (2010) A. C. Sewell et al. CLINICAL GENETICS
- Clinical variability in Sanfilippo B disease: a report on six patients in two related sibships
- (2010) J. J. P. van de KAMP et al. CLINICAL GENETICS
- Genetic heterogeneity and clinical variability in the Sanfilippo syndrome (types A, B, and C)
- (2010) J. J. P. VAN DE Kamp et al. CLINICAL GENETICS
- THE NATURAL HISTORY OF THE SEVERE FORM OF HUNTER'S SYNDROME: A STUDY BASED ON 52 CASES
- (2010) I. D. Young et al. DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY
- Childhood onset of Scheie syndrome, the attenuated form of mucopolysaccharidosis I
- (2010) Janet A. Thomas et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Mucopolysaccharidosis type IIIB may predominantly present with an attenuated clinical phenotype
- (2010) Marlies J. Valstar et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Cognitive and neuroradiological improvement in three patients with attenuated MPS I treated by laronidase
- (2010) V. Valayannopoulos et al. MOLECULAR GENETICS AND METABOLISM
- Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI
- (2010) Sean Turbeville et al. MOLECULAR GENETICS AND METABOLISM
- Mucopolysaccharidosis VI
- (2010) Vassili Valayannopoulos et al. Orphanet Journal of Rare Diseases
- Natural history of Polish patients with mucopolysaccharidosis type VI
- (2010) Agnieszka Jurecka et al. Open Medicine
- Mutations and polymorphisms in GUSB gene in mucopolysaccharidosis VII (Sly Syndrome)
- (2009) Shunji Tomatsu et al. HUMAN MUTATION
- Sanfilippo syndrome type C: mutation spectrum in the heparan sulfate acetyl-CoA: α-glucosaminide N-acetyltransferase (HGSNAT) gene
- (2009) Matthew Feldhammer et al. HUMAN MUTATION
- Mortality and cause of death in mucopolysaccharidosis type II—a historical review based on data from the Hunter Outcome Survey (HOS)
- (2009) S. A. Jones et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Guidelines for the Management of Mucopolysaccharidosis Type I
- (2009) Ana Maria Martins et al. JOURNAL OF PEDIATRICS
- Bone Marrow Transplantation in Children with Hunter Syndrome: Outcome after 7 to 17 Years
- (2009) Nathalie Guffon et al. JOURNAL OF PEDIATRICS
- Treatment reduces or stabilizes brain imaging abnormalities in patients with MPS I and II
- (2009) Raymond Y. Wang et al. MOLECULAR GENETICS AND METABOLISM
- Multidisciplinary Management of Hunter Syndrome
- (2009) J. Muenzer et al. PEDIATRICS
- Protein Misfolding as an Underlying Molecular Defect in Mucopolysaccharidosis III Type C
- (2009) Matthew Feldhammer et al. PLoS One
- Methods for assessing neurodevelopment in lysosomal storage diseases and related disorders: a multidisciplinary perspective
- (2008) Holly R Martin et al. ACTA PAEDIATRICA
- The Mutation p.Ser298Pro in the sulphamidase gene (SGSH) is associated with a slowly progressive clinical phenotype in mucopolysaccharidosis type IIIA (Sanfilippo A Syndrome)
- (2008) Ann Meyer et al. HUMAN MUTATION
- Neurological findings in Hunter disease: Pathology and possible therapeutic effects reviewed
- (2008) S. Al Sawaf et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Sanfilippo syndrome: A mini-review
- (2008) M. J. Valstar et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Use of Enzyme Replacement Therapy (Laronidase) before Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis I: Experience in 18 Patients
- (2008) Robert F. Wynn et al. JOURNAL OF PEDIATRICS
- Recognition and Diagnosis of Mucopolysaccharidosis II (Hunter Syndrome)
- (2008) R. Martin et al. PEDIATRICS
- Long-term Efficacy and Safety of Laronidase in the Treatment of Mucopolysaccharidosis I
- (2008) L. A. Clarke et al. PEDIATRICS
- Mucopolysaccharidosis I: Management and Treatment Guidelines
- (2008) J. Muenzer et al. PEDIATRICS
- Psychological status of patients with mucopolysaccharidosis type II and their parents
- (2008) Izumi Kuratsubo et al. PEDIATRICS INTERNATIONAL
- Long-term follow-up of a girl with Maroteaux-Lamy syndrome after bone marrow transplantation
- (2008) Ching-Chia Wang et al. World Journal of Pediatrics
- Clinical and genetic spectrum of Sanfilippo type C (MPS IIIC) disease in The Netherlands
- (2007) G.J.G. Ruijter et al. MOLECULAR GENETICS AND METABOLISM
Create your own webinar
Interested in hosting your own webinar? Check the schedule and propose your idea to the Peeref Content Team.
Create NowBecome a Peeref-certified reviewer
The Peeref Institute provides free reviewer training that teaches the core competencies of the academic peer review process.
Get Started