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Attenuated mucopolysaccharidosis: are you missing this debilitating condition?
- Published in:
- 2012
- By:
- Publication type:
- Editorial
Therapy for the mucopolysaccharidoses.
- Published in:
- Rheumatology, 2011, v. 50, n. suppl_5, p. v49, doi. 10.1093/rheumatology/ker396
- By:
- Publication type:
- Article
Putting collagen back in the collagen vascular disorders.
- Published in:
- Rheumatology, 2011, v. 50, n. suppl_5, p. v60, doi. 10.1093/rheumatology/ker398
- By:
- Publication type:
- Article
Overview of the mucopolysaccharidoses.
- Published in:
- Rheumatology, 2011, v. 50, n. suppl_5, p. v4, doi. 10.1093/rheumatology/ker394
- By:
- Publication type:
- Article
Treatment of thoracolumbar kyphosis in patients with mucopolysaccharidosis type I: results of an international consensus procedure.
- Published in:
- 2019
- By:
- Publication type:
- journal article
Cardiac features and effects of enzyme replacement therapy in Taiwanese patients with Mucopolysaccharidosis IVA.
- Published in:
- 2018
- By:
- Publication type:
- journal article
Anesthetic care and perioperative complications of children with Morquio syndrome.
- Published in:
- Pediatric Anesthesia, 2012, v. 22, n. 9, p. 901, doi. 10.1111/j.1460-9592.2012.03904.x
- By:
- Publication type:
- Article
Novel Therapies.
- Published in:
- Current Medical Literature: Lysosomal Storage Disease, 2012, v. 10, n. 2, p. 57
- Publication type:
- Article
Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure.
- Published in:
- Current Medical Literature: Lysosomal Storage Disease, 2012, v. 10, n. 1, p. 28
- Publication type:
- Article
Improvement in the range of joint motion in seven patients with mucopolysaccharidosis type II during experimental gene expression-targeted isoflavone therapy (GET IT).
- Published in:
- Current Medical Literature: Lysosomal Storage Disease, 2012, v. 10, n. 1, p. 15
- Publication type:
- Article
Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.
- Published in:
- Current Medical Literature: Lysosomal Storage Disease, 2008, v. 8, n. 2, p. 78
- Publication type:
- Article
Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome.
- Published in:
- Current Medical Literature: Lysosomal Storage Disease, 2008, v. 8, n. 2, p. 68
- Publication type:
- Article
ENZYME REPLACEMENT THERAPY FOR MUCOPOLYSACCHARIDOSES.
- Published in:
- Current Medical Literature: Lysosomal Storage Disease, 2004, v. 4, n. 2, p. 29
- By:
- Publication type:
- Article
Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 8, p. 1298, doi. 10.1038/mt.2015.99
- By:
- Publication type:
- Article
Regression Trends & Treatment Effectiveness to Improve Quality of Life for a Pre-Adolescent Girl with MPS IIIA.
- Published in:
- Journal of Developmental & Physical Disabilities, 2018, v. 30, n. 4, p. 545, doi. 10.1007/s10882-018-9601-5
- By:
- Publication type:
- Article
Therapeutic Options for Mucopolysaccharidoses: Current and Emerging Treatments.
- Published in:
- Drugs, 2019, v. 79, n. 10, p. 1103, doi. 10.1007/s40265-019-01147-4
- By:
- Publication type:
- Article
Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy.
- Published in:
- 2018
- By:
- Publication type:
- journal article
Mucopolysaccharidosis IIIB and mild skeletal anomalies: coexistence of <italic>NAGLU</italic> and <italic>CYP26B1</italic> missense variations in the same patient in a Chinese family.
- Published in:
- BMC Medical Genetics, 2018, v. 19, n. 1, p. 1, doi. 10.1186/s12881-018-0562-4
- By:
- Publication type:
- Article
Does enzyme replacement therapy influence the ocular changes in type VI mucopolysaccharidosis?
- Published in:
- Graefe's Archive of Clinical & Experimental Ophthalmology, 2009, v. 247, n. 7, p. 975, doi. 10.1007/s00417-008-1030-1
- By:
- Publication type:
- Article
Hurler Syndrome: A report of three cases.
- Published in:
- International Archives of Integrated Medicine, 2015, v. 2, n. 6, p. 210
- By:
- Publication type:
- Article
Abstract- Poster.
- Published in:
- 2022
- Publication type:
- Abstract
Sanfilippo syndrome.
- Published in:
- Learning Disability Practice, 2019, v. 22, n. 4, p. 17, doi. 10.7748/ldp.22.4.17.s15
- By:
- Publication type:
- Article
Treatment for Rare Enzyme Disorder.
- Published in:
- JAMA: Journal of the American Medical Association, 2018, v. 319, n. 1, p. 14, doi. 10.1001/jama.2017.20058
- By:
- Publication type:
- Article
Quality of life in mucopolysaccharidoses: construction of a specific measure using the focus group technique.
- Published in:
- BMC Research Notes, 2018, v. 11, p. 1, doi. 10.1186/s13104-018-3157-4
- By:
- Publication type:
- Article
Unfavourable outcome after uneventful anaesthesia and surgery in a child with Hurler syndrome.
- Published in:
- 2017
- By:
- Publication type:
- Letter to the Editor
Enhancement of Drug Delivery: Enzyme-replacement Therapy for Murine Morquio A Syndrome.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 6, p. 1094, doi. 10.1038/mt.2010.32
- By:
- Publication type:
- Article
Targeting of the CNS in MPS-IH Using a Nonviral Transferrin-α-L-iduronidase Fusion Gene Product.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 8, p. 1459, doi. 10.1038/mt.2008.119
- By:
- Publication type:
- Article
Thrombocytopenia associated with galsulfase treatment.
- Published in:
- 2011
- By:
- Publication type:
- Case Study
Laronidase.
- Published in:
- BioDrugs, 2002, v. 16, n. 4, p. 316, doi. 10.2165/00063030-200216040-00009
- Publication type:
- Article
Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches.
- Published in:
- Italian Journal of Pediatrics, 2018, v. 44, n. 2, p. N.PAG, doi. 10.1186/s13052-018-0565-y
- By:
- Publication type:
- Article
Neurobehavioral phenotypes of neuronopathic mucopolysaccharidoses.
- Published in:
- Italian Journal of Pediatrics, 2018, v. 44, n. 2, p. N.PAG, doi. 10.1186/s13052-018-0561-2
- By:
- Publication type:
- Article
The role of imaging in the skeletal involvement of mucopolysaccharidoses.
- Published in:
- Italian Journal of Pediatrics, 2018, v. 44, n. 2, p. N.PAG, doi. 10.1186/s13052-018-0556-z
- By:
- Publication type:
- Article
ENT and mucopolysaccharidoses.
- Published in:
- Italian Journal of Pediatrics, 2018, v. 44, n. 2, p. N.PAG, doi. 10.1186/s13052-018-0555-0
- By:
- Publication type:
- Article
Newborn screening in mucopolysaccharidoses.
- Published in:
- Italian Journal of Pediatrics, 2018, v. 44, n. 2, p. N.PAG, doi. 10.1186/s13052-018-0552-3
- By:
- Publication type:
- Article
Mucopolysaccharidoses: early diagnostic signs in infants and children.
- Published in:
- Italian Journal of Pediatrics, 2018, v. 44, n. 2, p. N.PAG, doi. 10.1186/s13052-018-0550-5
- By:
- Publication type:
- Article
The new frame for Mucopolysaccharidoses.
- Published in:
- 2018
- By:
- Publication type:
- Editorial
Caregivers' assessment of meaningful and relevant clinical outcome assessments for Sanfilippo syndrome.
- Published in:
- Journal of Patient-Reported Outcomes, 2022, v. 6, n. 1, p. 1, doi. 10.1186/s41687-022-00447-w
- By:
- Publication type:
- Article
Allogeneic blood SCT for children with Hurler's syndrome: results from the German multicenter approach MPS-HCT 2005.
- Published in:
- Bone Marrow Transplantation, 2009, v. 43, n. 5, p. 375, doi. 10.1038/bmt.2008.328
- By:
- Publication type:
- Article
Ocular manifestations and management recommendations of lysosomal storage disorders I: mucopolysaccharidoses.
- Published in:
- Clinical Ophthalmology, 2015, v. 9, p. 1633, doi. 10.2147/OPTH.S78368
- By:
- Publication type:
- Article
Anti-TNF-Alpha Therapy Enhances the Effects of Enzyme Replacement Therapy in Rats with Mucopolysaccharidosis Type VI.
- Published in:
- PLoS ONE, 2011, v. 6, n. 8, p. 1, doi. 10.1371/journal.pone.0022447
- By:
- Publication type:
- Article
Enzyme Replacement in a Human Model of Mucopolysaccharidosis IVA In Vitro and Its Biodistribution in the Cartilage of Wild Type Mice.
- Published in:
- PLoS ONE, 2010, v. 5, n. 8, p. 1, doi. 10.1371/journal.pone.0012194
- By:
- Publication type:
- Article
Miscellaneous.
- Published in:
- Current Medical Literature: Growth, Growth Hormone, & Metabolism, 2010, v. 3, n. 1, p. 19
- Publication type:
- Article
Transplantation of human umbilical mesenchymal stem cells cures the corneal defects of mucopolysaccharidosis VII mice.
- Published in:
- Stem Cells, 2013, v. 31, n. 10, p. 2116, doi. 10.1002/stem.1481
- By:
- Publication type:
- Article
Mucopolysaccharidosis: A broad review.
- Published in:
- 2022
- By:
- Publication type:
- journal article
Combined spinal arthrodesis with instrumentation for the management of progressive thoracolumbar kyphosis in children with mucopolysaccharidosis.
- Published in:
- European Spine Journal, 2014, v. 23, n. 12, p. 2751, doi. 10.1007/s00586-014-3186-1
- By:
- Publication type:
- Article
Sanfilippo syndrome: Overall review.
- Published in:
- Pediatrics International, 2015, v. 57, n. 3, p. 331, doi. 10.1111/ped.12636
- By:
- Publication type:
- Article
Anesthetic Management in Mucopolysaccharidoses.
- Published in:
- ISRN Anesthesiology, 2013, p. 1, doi. 10.1155/2013/791983
- By:
- Publication type:
- Article
Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure.
- Published in:
- 2011
- By:
- Publication type:
- journal article
Current and potential therapeutic strategies for mucopolysaccharidoses.
- Published in:
- Journal of Clinical Pharmacy & Therapeutics, 2014, v. 39, n. 3, p. 215, doi. 10.1111/jcpt.12136
- By:
- Publication type:
- Article
Anterior Hypopituitarism and Treatment Response in Hunter Syndrome: A Comparison of Two Patients.
- Published in:
- 2016
- By:
- Publication type:
- Case Study