Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement
Published 2021 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement
Authors
Keywords
-
Journal
Biomolecules
Volume 11, Issue 2, Pages 189
Publisher
MDPI AG
Online
2021-01-29
DOI
10.3390/biom11020189
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial
- (2020) Bernhard Gentner et al. BLOOD
- Progressive eye pathology in mucopolysaccharidosis type I mice and effects of enzyme replacement therapy
- (2020) Esteban A. Gonzalez et al. CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY
- Pulmonary involvement in selected lysosomal storage diseases and the impact of enzyme replacement therapy: A state‐of‐the art review
- (2020) Aleksandra Jezela‐Stanek et al. Clinical Respiratory Journal
- Preclinical development of SIG-005 for treatment of MPS I
- (2020) Marissa Donovan et al. MOLECULAR GENETICS AND METABOLISM
- Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations
- (2020) Rossella Parini et al. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
- Neonatal combination therapy improves some of the clinical manifestations in the Mucopolysaccharidosis type I murine model
- (2020) Ludovica Santi et al. MOLECULAR GENETICS AND METABOLISM
- Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I
- (2020) Keiko Miyadera et al. MOLECULAR THERAPY
- Hearing loss in patients with mucopolysaccharidoses ‐1 and ‐6 after hematopoietic cell transplantation: A longitudinal analysis
- (2020) Brigitte T. A. Broek et al. JOURNAL OF INHERITED METABOLIC DISEASE
- The evolution of pulmonary function in childhood onset Mucopolysaccharidosis type I
- (2020) A. Broomfield et al. MOLECULAR GENETICS AND METABOLISM
- Hurdles in treating Hurler disease: potential routes to achieve a “real” cure
- (2020) Brigitte T. A. van den Broek et al. Blood Advances
- Has resveratrol a potential for mucopolysaccharidosis treatment?
- (2020) Estera Rintz et al. EUROPEAN JOURNAL OF PHARMACOLOGY
- Ophthalmologic manifestations in Taiwanese patients with mucopolysaccharidoses
- (2019) Hsiang-Yu Lin et al. Molecular Genetics & Genomic Medicine
- Strategies for induction of immune tolerance to enzyme replacement therapy in Mucopolysaccharidosis Type I
- (2019) Arunabha Ghosh et al. Molecular Therapy-Methods & Clinical Development
- Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes
- (2019) Julie B. Eisengart et al. GENETICS IN MEDICINE
- Safe and sustained expression of human iduronidase after adeno-associated virus 9 intrathecal administration in infant rhesus monkeys
- (2019) Juliette Hordeaux et al. HUMAN GENE THERAPY
- Airway management in patients with mucopolysaccharidoses: The progression toward difficult intubation
- (2019) Lauren U. Madoff et al. PEDIATRIC ANESTHESIA
- Obstructive sleep apnea and craniofacial appearance in MPS type I-Hurler children after hematopoietic stem cell transplantation
- (2019) Till Koehne et al. Sleep and Breathing
- 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
- 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
- Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
- (2019) Natalia Gomez-Ospina et al. Nature Communications
- Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes
- (2019) Troy C. Lund et al. Scientific Reports
- Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice
- (2019) Roselena Silvestri Schuh et al. GENE THERAPY
- Children with mucopolysaccharidosis risk progressive visual dysfunction despite haematopoietic stem cell transplants
- (2018) Kristina Teär Fahnehjelm et al. ACTA PAEDIATRICA
- Salivary α-Iduronidase Activity as a Potential New Biomarker for the Diagnosis and Monitoring the Effect of Therapy in Mucopolysaccharidosis Type I
- (2018) Jaap van Doorn et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison
- (2018) Julie B Eisengart et al. GENETICS IN MEDICINE
- Peripherally derived macrophages can engraft the brain independent of irradiation and maintain an identity distinct from microglia
- (2018) James C. Cronk et al. JOURNAL OF EXPERIMENTAL MEDICINE
- RTB lectin-mediated delivery of lysosomal α- l -iduronidase mitigates disease manifestations systemically including the central nervous system
- (2018) Li Ou et al. MOLECULAR GENETICS AND METABOLISM
- Failure to shorten the diagnostic delay in two ultra-orphan diseases (mucopolysaccharidosis types I and III): potential causes and implications
- (2018) Gé-Ann Kuiper et al. Orphanet Journal of Rare Diseases
- Intrathecal/Intracerebroventricular enzyme replacement therapy for the mucopolysaccharidoses: efficacy, safety, and prospects
- (2018) Roberto Giugliani et al. Expert Opinion on Orphan Drugs
- Variability in the ocular phenotype in mucopolysaccharidosis
- (2018) Krishanthy Sornalingam et al. BRITISH JOURNAL OF OPHTHALMOLOGY
- Hematopoietic cell transplantation for severe MPS I in the first six months of life: The heart of the matter
- (2018) Elizabeth Braunlin et al. MOLECULAR GENETICS AND METABOLISM
- Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial
- (2018) Torayuki Okuyama et al. MOLECULAR THERAPY
- ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome
- (2018) Li Ou et al. MOLECULAR THERAPY
- Prolonged Expression of Secreted Enzymes in Dogs After Liver-Directed Delivery of Sleeping Beauty Transposons: Implications for Non-Viral Gene Therapy of Systemic Disease
- (2017) Elena L. Aronovich et al. HUMAN GENE THERAPY
- Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I
- (2017) Lalitha R. Belur et al. HUMAN GENE THERAPY
- 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
- 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
- Long term survival and cardiopulmonary outcome in children with Hurler syndrome after haematopoietic stem cell transplantation
- (2017) Su Han Lum et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Incomplete biomarker response in mucopolysaccharidosis type I after successful hematopoietic cell transplantation
- (2017) Gé-Ann Kuiper et al. MOLECULAR GENETICS AND METABOLISM
- Neonatal umbilical cord blood transplantation halts skeletal disease progression in the murine model of MPS-I
- (2017) Isabella Azario et al. Scientific Reports
- Allogeneic Hematopoietic Stem Cell Transplantation in Thirty-Four Pediatric Cases of Mucopolysaccharidosis—A Ten-Year Report from the China Children Transplant Group
- (2016) Jianmin Wang et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- Late Effects after Umbilical Cord Blood Transplantation in Very Young Children after Busulfan-Based, Myeloablative Conditioning
- (2016) Heather Allewelt et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- 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
- A novel explanation of corneal clouding in a bone marrow transplant-treated patient with Hurler syndrome
- (2016) Ching Yuan et al. EXPERIMENTAL EYE RESEARCH
- Progression of Hip Dysplasia in Mucopolysaccharidosis Type I Hurler After Successful Hematopoietic Stem Cell Transplantation
- (2016) Eveline J. Langereis et al. JOURNAL OF BONE AND JOINT SURGERY-AMERICAN VOLUME
- Treatment with pentosan polysulphate in patients with MPS I: results from an open label, randomized, monocentric phase II study
- (2016) Julia B. Hennermann et al. JOURNAL OF INHERITED METABOLIC DISEASE
- 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
- 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
- Insulin Receptor Antibody−α-N-Acetylglucosaminidase Fusion Protein Penetrates the Primate Blood–Brain Barrier and Reduces Glycosoaminoglycans in Sanfilippo Type B Fibroblasts
- (2016) Ruben J. Boado et al. MOLECULAR PHARMACEUTICS
- Neurodevelopmental Outcome after Hematopoietic Cell Transplantation in Inborn Errors of Metabolism: Current Considerations and Future Perspectives
- (2016) Peter van Hasselt et al. NEUROPEDIATRICS
- Musculoskeletal manifestations in mucopolysaccharidosis type I (Hurler syndrome) following hematopoietic stem cell transplantation
- (2016) Mona Schmidt et al. Orphanet Journal of Rare Diseases
- Pentosan Polysulfate: Oral Versus Subcutaneous Injection in Mucopolysaccharidosis Type I Dogs
- (2016) Calogera M. Simonaro et al. PLoS One
- The impact of laronidase treatment in otolaryngological manifestations of patients with mucopolysaccharidosis
- (2016) Ana Paula Fiuza Funicello Dualibi et al. Brazilian Journal of Otorhinolaryngology
- Hematopoietic Cell Transplantation for Mucopolysaccharidosis Patients Is Safe and Effective: Results after Implementation of International Guidelines
- (2015) Mieke Aldenhoven et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study
- (2015) M. Aldenhoven et al. BLOOD
- Busulfan fludarabine vs busulfan cyclophosphamide as a preparative regimen before allogeneic hematopoietic cell transplantation: systematic review and meta-analysis
- (2015) S Ben-Barouch et al. BONE MARROW TRANSPLANTATION
- Impaired bone remodeling and its correction by combination therapy in a mouse model of mucopolysaccharidosis-I
- (2015) Sonja C. Kuehn et al. HUMAN MOLECULAR GENETICS
- Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment
- (2015) Elsa G. Shapiro et al. MOLECULAR GENETICS AND METABOLISM
- Obstructive sleep apnea syndrome after hematopoietic stem cell transplantation in children with mucopolysaccharidosis type I
- (2015) Johan Moreau et al. MOLECULAR GENETICS AND METABOLISM
- Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I
- (2015) Patricia I. Dickson et al. MOLECULAR GENETICS AND METABOLISM
- Therapies for the bone in mucopolysaccharidoses
- (2015) Shunji Tomatsu et al. MOLECULAR GENETICS AND METABOLISM
- Biomarker responses correlate with antibody status in mucopolysaccharidosis type I patients on long-term enzyme replacement therapy
- (2015) Eveline J. Langereis 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
- 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
- Effects of Enzyme Replacement Therapy Started Late in a Murine Model of Mucopolysaccharidosis Type I
- (2015) Gabriela Pasqualim et al. PLoS One
- Early treatment is associated with improved cognition in Hurler syndrome
- (2014) Michele D. Poe et al. ANNALS OF NEUROLOGY
- Conditioning regimens for hematopoietic cell transplantation: one size does not fit all
- (2014) B. Gyurkocza 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
- Heparan Sulfate Inhibits Hematopoietic Stem and Progenitor Cell Migration and Engraftment in Mucopolysaccharidosis I
- (2014) H. Angharad Watson et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Effects of Neonatal Enzyme Replacement Therapy and Simvastatin Treatment on Cervical Spine Disease in Mucopolysaccharidosis I Dogs
- (2014) Joseph A Chiaro et al. JOURNAL OF BONE AND MINERAL RESEARCH
- Normalization and Improvement of CNS Deficits in Mice With Hurler Syndrome After Long-term Peripheral Delivery of BBB-targeted Iduronidase
- (2014) Salim S El-Amouri et al. MOLECULAR THERAPY
- Perioperative complications in patients diagnosed with mucopolysaccharidosis and the impact of enzyme replacement therapy followed by hematopoietic stem cell transplantation at early age
- (2014) Johanna H.A.M. Megens et al. PEDIATRIC ANESTHESIA
- Hematopoietic stem cell transplantation effects on spinal cord compression in Hurler
- (2014) Giovanna Ferrara et al. PEDIATRIC TRANSPLANTATION
- Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I
- (2014) C. Hinderer et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Thoracolumbar Kyphosis in Treated Mucopolysaccharidosis 1 (Hurler Syndrome)
- (2014) M. Naveed Yasin et al. SPINE
- Fludarabine and Exposure-Targeted Busulfan Compares Favorably with Busulfan/Cyclophosphamide-Based Regimens in Pediatric Hematopoietic Cell Transplantation: Maintaining Efficacy with Less Toxicity
- (2013) I.H. Bartelink et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning
- (2013) J. J. Boelens et al. BLOOD
- Ovarian function after allogeneic hematopoietic stem cell transplantation in childhood and adolescence
- (2013) A Vatanen et al. EUROPEAN JOURNAL OF ENDOCRINOLOGY
- Surgical management of thoracolumbar kyphosis in mucopolysaccharidosis type 1 in a reference center
- (2013) Kariman Abelin Genevois et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Hip Dysplasia in Patients With Hurler Syndrome (Mucopolysaccharidosis Type 1H)
- (2013) Dinesh P. Thawrani et al. JOURNAL OF PEDIATRIC ORTHOPAEDICS
- 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
- Long-term nonsense suppression therapy moderates MPS I-H disease progression
- (2013) Gwen Gunn et al. MOLECULAR GENETICS AND METABOLISM
- Early initiation of enzyme replacement therapy for the mucopolysaccharidoses
- (2013) Joseph Muenzer MOLECULAR GENETICS AND METABOLISM
- Standardization of α-L-iduronidase enzyme assay with Michaelis–Menten kinetics
- (2013) Li Ou et al. MOLECULAR GENETICS AND METABOLISM
- Osteoimmunology in mucopolysaccharidoses type I, II, VI and VII. Immunological regulation of the osteoarticular system in the course of metabolic inflammation
- (2013) V. Opoka-Winiarska et al. OSTEOARTHRITIS AND CARTILAGE
- Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood-brain barrier
- (2013) D. Wang et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Association Between Busulfan Exposure and Outcome in Children Receiving Intravenous Busulfan Before Hematopoietic Stem Cell Transplantation
- (2013) Marc Ansari et al. THERAPEUTIC DRUG MONITORING
- Fertility issues following hematopoietic stem cell transplantation
- (2013) André Tichelli et al. Expert Review of Hematology
- Improvements in Sensorineural Hearing Loss After Cord Blood Transplant in Patients With Mucopolysaccharidosis
- (2012) Victor Da Costa et al. ARCHIVES OF OTOLARYNGOLOGY-HEAD & NECK SURGERY
- Intracerebroventricular Transplantation of Human Bone Marrow-Derived Multipotent Progenitor Cells in an Immunodeficient Mouse Model of Mucopolysaccharidosis Type I (MPS-I)
- (2012) Zhenhong Nan et al. CELL TRANSPLANTATION
- Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy
- (2012) M. A. Saif et al. HAEMATOLOGICA
- 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
- 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
- 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
- A retrospective audit of anesthetic techniques and complications in children with mucopolysaccharidoses
- (2012) Geoff Frawley et al. PEDIATRIC ANESTHESIA
- 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
- Pharmaceutical therapies to recode nonsense mutations in inherited diseases
- (2012) Hui-Ling Rose Lee et al. PHARMACOLOGY & THERAPEUTICS
- Fertility after allogeneic haematopoietic stem cell transplantation in childhood and adolescence
- (2011) A Borgmann-Staudt et al. BONE MARROW TRANSPLANTATION
- 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
- Reversal of Lysosomal Storage in Brain of Adult MPS-I Mice with Intravenous Trojan Horse-Iduronidase Fusion Protein
- (2011) Ruben J. Boado et al. MOLECULAR PHARMACEUTICS
- Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I
- (2011) Daniel A. Wolf et al. NEUROBIOLOGY OF DISEASE
- 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
- Pathogenesis of skeletal and connective tissue involvement in the mucopolysaccharidoses: glycosaminoglycan storage is merely the instigator
- (2011) L. A. Clarke RHEUMATOLOGY
- Therapy for the mucopolysaccharidoses
- (2011) V. Valayannopoulos et al. RHEUMATOLOGY
- Orthopaedic aspects of mucopolysaccharidoses
- (2011) K. K. White RHEUMATOLOGY
- Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model
- (2010) I. Visigalli et al. BLOOD
- Diagnosis and management of ophthalmological features in patients with mucopolysaccharidosis
- (2010) S. Ferrari et al. BRITISH JOURNAL OF OPHTHALMOLOGY
- 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
- Airway Management in Children With Mucopolysaccharidoses
- (2009) Andrea H. Yeung et al. ARCHIVES OF OTOLARYNGOLOGY-HEAD & NECK SURGERY
- Short-term growth hormone treatment in children with Hurler syndrome after hematopoietic cell transplantation
- (2009) L E Polgreen et al. BONE MARROW TRANSPLANTATION
- The natural course and the impact of therapies of cardiac involvement in the mucopolysaccharidoses
- (2009) Vlasta Fesslová et al. CARDIOLOGY IN THE YOUNG
- Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy
- (2009) Robert F. Wynn 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
- Localized donor cells in brain of a Hunter disease patient after cord blood stem cell transplantation
- (2009) Ken Araya 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
- Systemic Correction of Storage Disease in MPS I NOD/SCID Mice Using the Sleeping Beauty Transposon System
- (2009) Elena L Aronovich et al. MOLECULAR THERAPY
- Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome
- (2009) D. Wang et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Progression of organ manifestations upon enzyme replacement therapy in a patient with mucopolysaccharidosis type I/Hurler
- (2009) Saadet Mercimek-Mahmutoglu et al. World Journal of Pediatrics
- Outcome in six children with mucopolysaccharidosis type IH, hurler syndrome, after haematopoietic stem cell transplantation (HSCT)
- (2008) Gunilla Malm et al. ACTA PAEDIATRICA
- Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression
- (2008) Maria-Veronica Munoz-Rojas et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART A
- Uptake of a Recombinant Human α-L-Iduronidase (laronidase) by Cultured Fibroblasts and Osteoblasts
- (2008) Takahiro Tsukimura et al. BIOLOGICAL & PHARMACEUTICAL BULLETIN
- Emerging trends in transplantation of inherited metabolic diseases
- (2008) V K Prasad et al. BONE MARROW TRANSPLANTATION
- Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation
- (2008) L E Polgreen et al. BONE MARROW TRANSPLANTATION
- Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I
- (2008) Patricia Dickson et al. JOURNAL OF CLINICAL INVESTIGATION
- A dose-optimization trial of laronidase (Aldurazyme®) in patients with mucopolysaccharidosis I
- (2008) Roberto Giugliani et al. MOLECULAR GENETICS AND METABOLISM
- Targeting of the CNS in MPS-IH Using a Nonviral Transferrin-α-l-iduronidase Fusion Gene Product
- (2008) Mark J Osborn et al. MOLECULAR THERAPY
- The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK
- (2008) David Moore et al. Orphanet Journal of Rare Diseases
- Long-term Efficacy and Safety of Laronidase in the Treatment of Mucopolysaccharidosis I
- (2008) L. A. Clarke et al. PEDIATRICS
- Mechanism of Glycosaminoglycan-Mediated Bone and Joint Disease
- (2007) Calogera M. Simonaro et al. AMERICAN JOURNAL OF PATHOLOGY
Find the ideal target journal for your manuscript
Explore over 38,000 international journals covering a vast array of academic fields.
SearchAdd your recorded webinar
Do you already have a recorded webinar? Grow your audience and get more views by easily listing your recording on Peeref.
Upload Now