Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations
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Title
Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations
Authors
Keywords
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Journal
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
Volume 21, Issue 8, Pages 2975
Publisher
MDPI AG
Online
2020-04-23
DOI
10.3390/ijms21082975
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Note: Only part of the references are listed.- Novel Enzyme Replacement Therapies for Neuropathic Mucopolysaccharidoses
- (2020) Yuji Sato et al. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
- Assessment of Activity of Daily Life in Mucopolysaccharidosis Type II Patients with Hematopoietic Stem Cell Transplantation
- (2020) Yasuyuki Suzuki et al. Diagnostics
- Strategies for induction of immune tolerance to enzyme replacement therapy in Mucopolysaccharidosis Type I
- (2019) Arunabha Ghosh et al. Molecular Therapy-Methods & Clinical Development
- Enzyme replacement therapy outcomes across the disease spectrum: findings from the mucopolysaccharidosis VI Clinical Surveillance Program
- (2019) Paul R Harmatz et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Early enzyme replacement therapy enables a successful hematopoietic stem cell transplantation in mucopolysaccharidosis type IH: Divergent clinical outcomes in two Japanese siblings
- (2019) Narutoshi Yamazaki et al. BRAIN & DEVELOPMENT
- Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and Future
- (2019) Madeleine Taylor et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- Vestronidase Alfa: A Review in Mucopolysaccharidosis VII
- (2019) Emma H. McCafferty et al. BIODRUGS
- Effect of enzyme replacement therapy on the growth of patients with Morquio A
- (2019) Caitlin Doherty et al. JOURNAL OF HUMAN GENETICS
- Treating Lysosomal Storage Disorders: What Have We Learnt?
- (2019) Robin H Lachmann JOURNAL OF INHERITED METABOLIC DISEASE
- Targeting a Pre-existing Anti-transgene T Cell Response for Effective Gene Therapy of MPS-I in the Mouse Model of the Disease
- (2019) Giorgia Squeri et al. MOLECULAR THERAPY
- Growth charts for patients with Sanfilippo syndrome (Mucopolysaccharidosis type III)
- (2019) Nicole M. Muschol et al. Orphanet Journal of Rare Diseases
- Long-term outcomes of enzyme replacement therapy for Taiwanese patients with Mucopolysaccharidosis I
- (2019) Hsiang-Yu Lin et al. Pediatrics and Neonatology
- Therapeutic Options for Mucopolysaccharidoses: Current and Emerging Treatments
- (2019) Kazuki Sawamoto et al. DRUGS
- Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation
- (2019) Lynda E. Polgreen et al. PEDIATRIC RESEARCH
- Ophthalmological Findings in Mucopolysaccharidoses
- (2019) Shizuka Tomatsu et al. Journal of Clinical Medicine
- Growth patterns for untreated individuals with MPS I: Report from the international MPS I registry
- (2019) David Viskochil et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART A
- Six-Minute Walk Test
- (2019) Steve Salzman et al. CHEST
- Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes
- (2019) Troy C. Lund et al. Scientific Reports
- Auditory Characteristics in Patients With Mucopolysaccharidosis
- (2019) Jungmin Ahn et al. OTOLOGY & NEUROTOLOGY
- Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison
- (2018) Julie B Eisengart et al. GENETICS IN MEDICINE
- Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study
- (2018) Christian J. Hendriksz et al. MOLECULAR GENETICS AND METABOLISM
- Growth impairment in mucopolysaccharidoses
- (2018) Melodie Melbouci et al. MOLECULAR GENETICS AND METABOLISM
- Glycosaminoglycans analysis in blood and urine of patients with mucopolysaccharidosis
- (2018) Shaukat A. Khan 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
- Safety, immunogenicity, and clinical outcomes in patients with Morquio A syndrome participating in 2 sequential open-label studies of elosulfase alfa enzyme replacement therapy (MOR-002/MOR-100), representing 5 years of treatment
- (2018) Christian Hendriksz et al. MOLECULAR GENETICS AND METABOLISM
- Efficacy of Idursulfase therapy in patients with Mucopolysaccharidosis type II who initiated enzyme replacement therapy in adult age. A systematic review of the literature
- (2018) J. Pérez-López et al. MOLECULAR GENETICS AND METABOLISM
- Immune modulation in a patient with Morquio syndrome treated with enzyme replacement therapy
- (2018) Angela Sun et al. Journal of Allergy and Clinical Immunology-In Practice
- Variability in the ocular phenotype in mucopolysaccharidosis
- (2018) Krishanthy Sornalingam et al. BRITISH JOURNAL OF OPHTHALMOLOGY
- Aortic root dilatation in patients with mucopolysaccharidoses and the impact of enzyme replacement therapy
- (2018) Fabiano de Oliveira Poswar et al. HEART AND VESSELS
- Hunter syndrome: Long-term idursulfase treatment does not protect patients against DNA oxidation and cytogenetic damage
- (2018) Carlos Eduardo Diaz Jacques et al. MUTATION RESEARCH-GENETIC TOXICOLOGY AND ENVIRONMENTAL MUTAGENESIS
- Cardiac features and effects of enzyme replacement therapy in Taiwanese patients with Mucopolysaccharidosis IVA
- (2018) Hsiang-Yu Lin et al. Orphanet Journal of Rare Diseases
- Long-term effects of enzyme replacement therapy for Taiwanese patients with mucopolysaccharidosis IVA
- (2018) Hsiang-Yu Lin et al. Pediatrics and Neonatology
- Substrate accumulation and extracellular matrix remodelling promote persistent upper airway disease in mucopolysaccharidosis patients on enzyme replacement therapy
- (2018) Abhijit Ricky Pal et al. PLoS One
- The relationships between urinary glycosaminoglycan levels and phenotypes of mucopolysaccharidoses
- (2018) Hsiang-Yu Lin et al. Molecular Genetics & Genomic Medicine
- 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
- The role of innate immunity in mucopolysaccharide diseases
- (2018) Helen Parker et al. JOURNAL OF NEUROCHEMISTRY
- Vessel shape alterations of the vertebrobasilar arteries in Mucopolysaccharidosis type IVa (Morquio A) patients
- (2017) Yasemin Tanyildizi et al. EUROPEAN JOURNAL OF RADIOLOGY
- The Carotid Intima-Media Thickness and Arterial Stiffness of Pediatric Mucopolysaccharidosis Patients Are Increased Compared to Both Pediatric and Adult Controls
- (2017) Raymond Wang et al. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
- The Frequency of Carpal Tunnel Syndrome in Hurler Syndrome After Peritransplant Enzyme Replacement Therapy: A Retrospective Comparison
- (2017) Mitchell L. Wyffels et al. JOURNAL OF HAND SURGERY-AMERICAN VOLUME
- Correlation between urinary GAG and anti-idursulfase ERT neutralizing antibodies during treatment with NICIT immune tolerance regimen: A case report
- (2017) Sarah Kim et al. MOLECULAR GENETICS AND METABOLISM
- Ten plus one challenges in diseases of the lysosomal system
- (2017) Gregory A. Grabowski et al. MOLECULAR GENETICS AND METABOLISM
- Efficacy of laronidase therapy in patients with mucopolysaccharidosis type I who initiated enzyme replacement therapy in adult age. A systematic review and meta-analysis
- (2017) Jordi Pérez-López et al. MOLECULAR GENETICS AND METABOLISM
- The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
- (2017) P. Harmatz 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
- Early disease progression of Hurler syndrome
- (2017) Bridget T. Kiely et al. Orphanet Journal of Rare Diseases
- Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS)
- (2017) Joseph Muenzer et al. Orphanet Journal of Rare Diseases
- Successful desensitization of elosulfase alfa–induced anaphylaxis in a pediatric patient with Morquio syndrome
- (2017) Hakan Guvenir et al. Journal of Allergy and Clinical Immunology-In Practice
- Efficacy and safety of intravenous laronidase for mucopolysaccharidosis type I: A systematic review and meta-analysis
- (2017) Alícia Dorneles Dornelles et al. PLoS One
- Surgical Reconstruction for Severe Tracheal Obstruction in Morquio A Syndrome
- (2016) Christian Pizarro et al. ANNALS OF THORACIC SURGERY
- 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
- Short Communication Impact of early enzyme-replacement therapy for mucopolysaccharidosis VI: results of a long-term follow-up of Brazilian siblings
- (2016) J.F. Franco et al. GENETICS AND MOLECULAR RESEARCH
- IgE-Mediated Hypersensitivity and Desensitisation with Recombinant Enzymes in Pompe Disease and Type I and Type VI Mucopolysaccharidosis
- (2016) Murat Capanoglu et al. INTERNATIONAL ARCHIVES OF ALLERGY AND IMMUNOLOGY
- Tracheobronchial stents in mucopolysaccharidosis
- (2016) Reiter Karl et al. INTERNATIONAL JOURNAL OF PEDIATRIC OTORHINOLARYNGOLOGY
- Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome
- (2016) Christian J. Hendriksz et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Clinical course of sly syndrome (mucopolysaccharidosis type VII)
- (2016) Adriana M Montaño et al. JOURNAL OF MEDICAL GENETICS
- Outcomes of Long-Term Treatment with Laronidase in Patients with Mucopolysaccharidosis Type I
- (2016) Sarah Laraway 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
- Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome
- (2016) Christian J. Hendriksz et al. MOLECULAR GENETICS AND METABOLISM
- The natural history of growth in patients with Hunter syndrome: Data from the Hunter Outcome Survey (HOS)
- (2016) Rossella Parini et al. MOLECULAR GENETICS AND METABOLISM
- Obstructive airway in Morquio A syndrome, the past, the present and the future
- (2016) Shunji Tomatsu et al. MOLECULAR GENETICS AND METABOLISM
- Cardiac structure and function and effects of enzyme replacement therapy in patients with mucopolysaccharidoses I, II, IVA and VI
- (2016) Hsiang-Yu Lin et al. MOLECULAR GENETICS AND METABOLISM
- Effect of anti-laronidase antibodies on efficacy and safety of laronidase enzyme replacement therapy for MPS I: A comprehensive meta-analysis of pooled data from multiple studies
- (2016) Yong Xue et al. MOLECULAR GENETICS AND METABOLISM
- Health-related quality of life in mucopolysaccharidosis: looking beyond biomedical issues
- (2016) Christian J. Hendriksz et al. Orphanet Journal of Rare Diseases
- Severe tracheal and bronchial collapse in adults with type II mucopolysaccharidosis
- (2016) M. Rutten et al. Orphanet Journal of Rare Diseases
- The impact of laronidase treatment in otolaryngological manifestations of patients with mucopolysaccharidosis
- (2016) Ana Paula Fiuza Funicello Dualibi et al. Brazilian Journal of Otorhinolaryngology
- Diagnosis and management of infusion-related hypersensitivity reactions to enzyme replacement therapy for lysosomal diseases: The role of desensitization
- (2016) Carolina S. Aranda et al. Journal of Allergy and Clinical Immunology-In Practice
- Heart and Cardiovascular Involvement in Patients with Mucopolysaccharidosis Type IVA (Morquio-A Syndrome)
- (2016) Christoph Kampmann et al. PLoS One
- Safety and physiological effects of two different doses of elosulfase alfa in patients with morquio a syndrome: A randomized, double-blind, pilot study
- (2015) Barbara K. Burton et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART A
- Oxidative stress and inflammation in mucopolysaccharidosis type IVA patients treated with enzyme replacement therapy
- (2015) Bruna Donida et al. BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE
- Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III Trial
- (2015) Becky Schweighardt et al. CLINICAL THERAPEUTICS
- Enzyme Replacement Therapies and Immunogenicity in Lysosomal Storage Diseases: Is There a Pattern?
- (2015) Paul Harmatz CLINICAL THERAPEUTICS
- Alterations of mucosa of the larynx and hypopharynx in patients with mucopolysaccharidoses
- (2015) A Keilmann et al. JOURNAL OF LARYNGOLOGY AND OTOLOGY
- Activities of daily living in patients with Hunter syndrome: Impact of enzyme replacement therapy and hematopoietic stem cell transplantation
- (2015) Julian Tanjuakio et al. MOLECULAR GENETICS AND METABOLISM
- Multi-domain impact of elosulfase alfa in Morquio A syndrome in the pivotal phase III trial
- (2015) Christian J. Hendriksz et al. MOLECULAR GENETICS AND METABOLISM
- Enzyme replacement therapy in newborn mucopolysaccharidosis IVA mice: Early treatment rescues bone lesions?
- (2015) Shunji Tomatsu et al. MOLECULAR GENETICS AND METABOLISM
- Safety and efficacy of enzyme replacement therapy with idursulfase beta in children aged younger than 6years with Hunter syndrome
- (2015) Young Bae Sohn et al. MOLECULAR GENETICS AND METABOLISM
- Therapies for the bone in mucopolysaccharidoses
- (2015) Shunji Tomatsu et al. MOLECULAR GENETICS AND METABOLISM
- Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome
- (2015) Abhijit Ricky Pal et al. Orphanet Journal of Rare Diseases
- Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome)
- (2015) Arian Pano et al. Orphanet Journal of Rare Diseases
- 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
- Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome
- (2015) Shunji Tomatsu et al. Drug Design Development and Therapy
- 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
- Airway-Related Symptoms and Surgeries in Patients With Mucopolysaccharidosis I
- (2014) Pamela Arn et al. ANNALS OF OTOLOGY RHINOLOGY AND LARYNGOLOGY
- Neonatal bone marrow transplantation prevents bone pathology in a mouse model of mucopolysaccharidosis type I
- (2014) A. Pievani et al. BLOOD
- 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
- Mucopolysaccharidosis: Otolaryngologic findings, obstructive sleep apnea and accumulation of glucosaminoglycans in lymphatic tissue of the upper airway
- (2014) Burhanettin Gönüldaş et al. INTERNATIONAL JOURNAL OF PEDIATRIC OTORHINOLARYNGOLOGY
- Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study
- (2014) Christian J. Hendriksz et al. JOURNAL OF INHERITED METABOLIC DISEASE
- 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
- Impact of Enzyme Replacement Therapy on Linear Growth in Korean Patients with Mucopolysaccharidosis Type II (Hunter Syndrome)
- (2014) Sung Yoon Cho et al. JOURNAL OF KOREAN MEDICAL SCIENCE
- CT and endoscopic evaluation of larynx and trachea in mucopolysaccharidoses
- (2014) Noriko Morimoto et al. MOLECULAR GENETICS AND METABOLISM
- 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
- Laronidase hypersensitivity and desensitization in type I mucopolysaccharidosis: a case report
- (2014) Luis Felipe Ensina et al. PEDIATRIC ALLERGY AND IMMUNOLOGY
- Cytogenetic biomonitoring in mucopolyssacharosis I, II and IV patients treated with enzyme replacement therapy
- (2014) Joice Marques Guilheiro et al. TOXICOLOGY MECHANISMS AND METHODS
- IgE-mediated anaphylaxis and allergic reactions to idursulfase in patients with Hunter syndrome
- (2013) J. Kim et al. ALLERGY
- 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
- Does early use of enzyme replacement therapy alter the natural history of mucopolysaccharidosis I? Experience in three siblings
- (2013) Sarah Laraway et al. MOLECULAR GENETICS AND METABOLISM
- The effect of idursulfase on growth in patients with Hunter syndrome: Data from the Hunter Outcome Survey (HOS)
- (2013) Simon A. Jones et al. MOLECULAR GENETICS AND METABOLISM
- Enzyme replacement therapy with galsulfase in 34 children younger than five years of age with MPS VI
- (2013) Dafne D.G. Horovitz et al. MOLECULAR GENETICS AND METABOLISM
- Enzyme replacement therapy started at birth improves outcome in difficult-to-treat organs in mucopolysaccharidosis I mice
- (2013) Guilherme Baldo et al. MOLECULAR GENETICS AND METABOLISM
- The relationship between anti-idursulfase antibody status and safety and efficacy outcomes in attenuated mucopolysaccharidosis II patients aged 5years and older treated with intravenous idursulfase
- (2013) A.J. Barbier et al. MOLECULAR GENETICS AND METABOLISM
- Up to five years experience with 11 mucopolysaccharidosis type VI patients
- (2013) Marion M.M.G. Brands 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
- Review of clinical presentation and diagnosis of mucopolysaccharidosis IVA
- (2013) C.J. Hendriksz et al. MOLECULAR GENETICS AND METABOLISM
- Haploidentical stem cell transplantation in two children with mucopolysaccharidosis VI: clinical and biochemical outcome
- (2013) Sandra Jester et al. Orphanet Journal of Rare Diseases
- Phase I/II clinical trial of enzyme replacement therapy with idursulfase beta in patients with mucopolysaccharidosis II (Hunter Syndrome)
- (2013) Young Bae Sohn et al. Orphanet Journal of Rare Diseases
- Characterization of pulmonary function impairments in patients with mucopolysaccharidoses-changes with age and treatment
- (2013) Shuan-Pei Lin et al. PEDIATRIC PULMONOLOGY
- Enzyme replacement therapy improves joint motion and outcome of the 12-min walk test in a mucopolysaccharidosis type VI patient previously treated with bone marrow transplantation
- (2012) Young Bae Sohn et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART A
- Mucopolysaccharidosis: Cardiologic features and effects of enzyme-replacement therapy in 24 children with MPS I, II and VI
- (2012) Marion M. M. G. Brands et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy
- (2012) E. Braunlin et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Respiratory and sleep disorders in mucopolysaccharidosis
- (2012) Kenneth I. Berger et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Management of mucopolysaccharidosis type IH (Hurler’s syndrome) presenting in infancy with severe dilated cardiomyopathy: a single institution’s experience
- (2012) Daniel H. Wiseman et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Plasma and urinary levels of dermatan sulfate and heparan sulfate derived disaccharides after long-term enzyme replacement therapy (ERT) in MPS I: correlation with the timing of ERT and with total urinary excretion of glycosaminoglycans
- (2012) Minke H. de Ru et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Effect of rapid cessation of enzyme replacement therapy: A report of 5 cases and a review of the literature
- (2012) Agnieszka Jurecka 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
- Mucopolysaccharidosis type I (Hurler syndrome) and anesthesia: the impact of bone marrow transplantation, enzyme replacement therapy, and fiberoptic intubation on airway management
- (2012) Katherine Kirkpatrick et al. PEDIATRIC ANESTHESIA
- Clinical guidelines for diagnosing and managing ocular manifestations in children with mucopolysaccharidosis
- (2011) Kristina T. Fahnehjelm et al. ACTA OPHTHALMOLOGICA
- 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
- Retrospective analysis of the clinical manifestations and survival of Korean patients with mucopolysaccharidosis type II: Emphasis on the cardiovascular complication and mortality cases
- (2011) Young Bae Sohn et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART A
- The role of enzyme replacement therapy in severe Hunter syndrome—an expert panel consensus
- (2011) Joseph Muenzer et al. EUROPEAN JOURNAL OF PEDIATRICS
- Idursulfase treatment of Hunter syndrome in children younger than 6 years: Results from the Hunter Outcome Survey
- (2011) Joseph Muenzer et al. GENETICS IN MEDICINE
- Management of infusion-related reactions to enzyme replacement therapy in a cohort of patients with mucopolysaccharidosis disorders
- (2011) E. Miebach INTERNATIONAL JOURNAL OF CLINICAL PHARMACOLOGY AND THERAPEUTICS
- Teste de caminhada de seis minutos: uma ferramenta valiosa na avaliação do comprometimento pulmonar
- (2011) Jaime Eduardo Morales-Blanhir et al. Jornal Brasileiro de Pneumologia
- Hearing loss in patients with mucopolysaccharidosis II: Data from HOS – the Hunter Outcome Survey
- (2011) Annerose Keilmann et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management
- (2011) Elizabeth A. Braunlin et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Treatment of mucopolysaccharidosis type II (Hunter syndrome) with idursulfase: the relevance of clinical trial end points
- (2011) Emma Glamuzina et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Oxidative stress in patients with mucopolysaccharidosis type II before and during enzyme replacement therapy
- (2011) Letícia Filippon et al. MOLECULAR GENETICS AND METABOLISM
- Incidence and timing of infusion-related reactions in patients with mucopolysaccharidosis type II (Hunter syndrome) on idursulfase therapy in the real-world setting: A perspective from the Hunter Outcome Survey (HOS)
- (2011) Barbara K. Burton et al. MOLECULAR GENETICS AND METABOLISM
- DNA damage in leukocytes from pretreatment mucopolysaccharidosis type II patients; protective effect of enzyme replacement therapy
- (2011) Letícia Filippon et al. MUTATION RESEARCH-GENETIC TOXICOLOGY AND ENVIRONMENTAL MUTAGENESIS
- Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders
- (2011) Carla EM Hollak et al. Orphanet Journal of Rare Diseases
- Enzyme Replacement Therapy and/or Hematopoietic Stem Cell Transplantation at diagnosis in patients with Mucopolysaccharidosis type I: results of a European consensus procedure
- (2011) Minke H de Ru et al. Orphanet Journal of Rare Diseases
- Growth pattern and growth prediction of body height in children with mucopolysaccharidosis type II
- (2010) Agnieszka Rozdzynska et al. ACTA PAEDIATRICA
- Systemic therapies for mucopolysaccharidosis: ocular changes following haematopoietic stem cell transplantation or enzyme replacement therapy - a review
- (2010) C Gail Summers et al. CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY
- First experience of enzyme replacement therapy with idursulfase in Spanish patients with Hunter syndrome under 5 years of age: Case observations from the Hunter Outcome Survey (HOS)
- (2010) C. Alcalde-Martín et al. European Journal of Medical Genetics
- Importance of surgical history in diagnosing mucopolysaccharidosis type II (Hunter syndrome): Data from the Hunter Outcome Survey
- (2010) Nancy J. Mendelsohn et al. GENETICS IN MEDICINE
- Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome
- (2010) Joseph Muenzer et al. GENETICS IN MEDICINE
- Efficacy of recombinant human α-L-iduronidase (laronidase) on restricted range of motion of upper extremities in mucopolysaccharidosis type I patients
- (2010) Anna Tylki-Szymanska et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase
- (2010) Paul Harmatz et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI
- (2010) Sean Turbeville et al. MOLECULAR GENETICS AND METABOLISM
- Polysomnographic characteristics in patients with mucopolysaccharidoses
- (2010) Hsiang-Yu Lin et al. PEDIATRIC PULMONOLOGY
- Laronidase for Cardiopulmonary Disease in Hurler Syndrome 12 Years After Bone Marrow Transplantation
- (2010) V. Valayannopoulos et al. PEDIATRICS
- The natural course and the impact of therapies of cardiac involvement in the mucopolysaccharidoses
- (2009) Vlasta Fesslová et al. CARDIOLOGY IN THE YOUNG
- Enzyme replacement therapy for mucopolysaccharidosis VI from 8 weeks of age-a sibling control study
- (2009) JJ McGill et al. CLINICAL GENETICS
- Mucopolysaccharidosis VI: the Italian experience
- (2009) Maurizio Scarpa et al. EUROPEAN JOURNAL OF PEDIATRICS
- Does enzyme replacement therapy influence the ocular changes in type VI mucopolysaccharidosis?
- (2009) Susanne Pitz et al. GRAEFES ARCHIVE FOR CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY
- Bone Marrow Transplantation in Children with Hunter Syndrome: Outcome after 7 to 17 Years
- (2009) Nathalie Guffon et al. JOURNAL OF PEDIATRICS
- Anthropometric data of 14 patients with mucopolysaccharidosis I: Retrospective analysis and efficacy of recombinant human α-l-iduronidase (laronidase)
- (2009) Anna Tylki-Szymanska et al. MOLECULAR GENETICS AND METABOLISM
- Japan Elaprase® Treatment (JET) study: Idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS II)
- (2009) Torayuki Okuyama et al. MOLECULAR GENETICS AND METABOLISM
- Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants
- (2009) Priya S. Kishnani et al. MOLECULAR GENETICS AND METABOLISM
- Lysosomal disorders: From storage to cellular damage
- (2008) Andrea Ballabio et al. BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH
- 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
- Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase
- (2008) Paul Harmatz et al. MOLECULAR GENETICS AND METABOLISM
- Long-term Efficacy and Safety of Laronidase in the Treatment of Mucopolysaccharidosis I
- (2008) L. A. Clarke et al. PEDIATRICS
- Reversed papilledema in an MPS VI patient with galsulfase (Naglazyme®) therapy
- (2008) Selim T. Koseoglu et al. INTERNATIONAL OPHTHALMOLOGY
- Mechanism of Glycosaminoglycan-Mediated Bone and Joint Disease
- (2007) Calogera M. Simonaro et al. AMERICAN JOURNAL OF PATHOLOGY
- Mutational and oxidative stress analysis in patients with mucopolysaccharidosis type I undergoing enzyme replacement therapy
- (2007) Vanessa Gonçalves Pereira et al. CLINICA CHIMICA ACTA
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