Works matching DE "MUCOPOLYSACCHARIDOSIS treatment"
Results: 69
Sanfilippo syndrome.
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- Learning Disability Practice, 2019, v. 22, n. 4, p. 17, doi. 10.7748/ldp.22.4.17.s15
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- Article
Allogeneic blood SCT for children with Hurler's syndrome: results from the German multicenter approach MPS-HCT 2005.
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- Bone Marrow Transplantation, 2009, v. 43, n. 5, p. 375, doi. 10.1038/bmt.2008.328
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- Article
Carpal tunnel syndrome in mucopolysaccharidosis: the value of rare disease registries.
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- 2017
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- Publication type:
- journal article
Anaesthetic implications of the changing management of patients with mucopolysaccharidosis.
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- 2016
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- Publication type:
- journal article
Mucopolysaccharidosis VI, a Case Report.
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- Journal of Guilan University of Medical Sciences, 2014, v. 22, n. 89, p. 83
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- Article
Anesthetic Management in Mucopolysaccharidoses.
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- ISRN Anesthesiology, 2013, p. 1, doi. 10.1155/2013/791983
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- Article
Drug Repurposing Patent Applications April-June 2015.
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- Assay & Drug Development Technologies, 2015, v. 13, n. 10, p. 654, doi. 10.1089/adt.2015.29030.pq2
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- Article
Treatment of MPS VII (Sly disease) by allogeneic BMT in a female with homozygous A619V mutation.
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- Bone Marrow Transplantation, 1998, v. 21, n. 6, p. 629, doi. 10.1038/sj.bmt.1701141
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- Article
Expensive drugs for rare disorders: to treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI.
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- Current Medical Research & Opinion, 2009, v. 25, n. 5, p. 1285, doi. 10.1185/03007990902892633
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- Article
Sanfilippo syndrome: Overall review.
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- Pediatrics International, 2015, v. 57, n. 3, p. 331, doi. 10.1111/ped.12636
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- Article
Treatment for Rare Enzyme Disorder.
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- JAMA: Journal of the American Medical Association, 2018, v. 319, n. 1, p. 14, doi. 10.1001/jama.2017.20058
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- Article
Pentosan Polysulfate: Oral Versus Subcutaneous Injection in Mucopolysaccharidosis Type I Dogs.
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- PLoS ONE, 2016, v. 11, n. 4, p. 1, doi. 10.1371/journal.pone.0153136
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- Article
Novel Therapies.
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- Current Medical Literature: Lysosomal Storage Disease, 2012, v. 10, n. 2, p. 57
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- Article
Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure.
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- Current Medical Literature: Lysosomal Storage Disease, 2012, v. 10, n. 1, p. 28
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- Article
Improvement in the range of joint motion in seven patients with mucopolysaccharidosis type II during experimental gene expression-targeted isoflavone therapy (GET IT).
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- Current Medical Literature: Lysosomal Storage Disease, 2012, v. 10, n. 1, p. 15
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- Article
Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.
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- Current Medical Literature: Lysosomal Storage Disease, 2008, v. 8, n. 2, p. 78
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- Article
Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome.
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- Current Medical Literature: Lysosomal Storage Disease, 2008, v. 8, n. 2, p. 68
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- Article
ENZYME REPLACEMENT THERAPY FOR MUCOPOLYSACCHARIDOSES.
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- Current Medical Literature: Lysosomal Storage Disease, 2004, v. 4, n. 2, p. 29
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- Article
Thrombocytopenia associated with galsulfase treatment.
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- 2011
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- Case Study
Attenuated mucopolysaccharidosis: are you missing this debilitating condition?
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- 2012
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- Publication type:
- Editorial
Therapy for the mucopolysaccharidoses.
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- Rheumatology, 2011, v. 50, n. suppl_5, p. v49, doi. 10.1093/rheumatology/ker396
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- Article
Putting collagen back in the collagen vascular disorders.
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- Rheumatology, 2011, v. 50, n. suppl_5, p. v60, doi. 10.1093/rheumatology/ker398
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- Article
Overview of the mucopolysaccharidoses.
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- Rheumatology, 2011, v. 50, n. suppl_5, p. v4, doi. 10.1093/rheumatology/ker394
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- Article
Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy.
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- 2018
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- Publication type:
- journal article
Combined spinal arthrodesis with instrumentation for the management of progressive thoracolumbar kyphosis in children with mucopolysaccharidosis.
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- European Spine Journal, 2014, v. 23, n. 12, p. 2751, doi. 10.1007/s00586-014-3186-1
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- Article
Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.
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- Molecular Therapy, 2015, v. 23, n. 8, p. 1298, doi. 10.1038/mt.2015.99
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- Article
Enhancement of Drug Delivery: Enzyme-replacement Therapy for Murine Morquio A Syndrome.
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- Molecular Therapy, 2010, v. 18, n. 6, p. 1094, doi. 10.1038/mt.2010.32
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- Article
Hurler Syndrome: A report of three cases.
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- International Archives of Integrated Medicine, 2015, v. 2, n. 6, p. 210
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- Article
Current and potential therapeutic strategies for mucopolysaccharidoses.
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- Journal of Clinical Pharmacy & Therapeutics, 2014, v. 39, n. 3, p. 215, doi. 10.1111/jcpt.12136
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- Article
A new case of mucopolysaccharidosis VII presenting as non immune hydrops fetalis.
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- European Journal of Pediatrics, 2003, v. 162, n. 7/8, p. 520, doi. 10.1007/s00431-003-1198-7
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- Article
Laronidase.
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- BioDrugs, 2002, v. 16, n. 4, p. 316, doi. 10.2165/00063030-200216040-00009
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- Article
NDST1 Preferred Promoter Confirmation and Identification of Corresponding Transcriptional Inhibitors as Substrate Reduction Agents for Multiple Mucopolysaccharidosis Disorders.
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- PLoS ONE, 2016, v. 11, n. 9, p. 1, doi. 10.1371/journal.pone.0162145
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- Publication type:
- Article
Heart and Cardiovascular Involvement in Patients with Mucopolysaccharidosis Type IVA (Morquio-A Syndrome).
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- PLoS ONE, 2016, v. 11, n. 9, p. 1, doi. 10.1371/journal.pone.0162612
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- Article
Unfavourable outcome after uneventful anaesthesia and surgery in a child with Hurler syndrome.
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- 2017
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- Publication type:
- Letter to the Editor
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.
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- BMC Medical Genetics, 2018, v. 19, n. 1, p. 1, doi. 10.1186/s12881-018-0562-4
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- Article
Caregivers' assessment of meaningful and relevant clinical outcome assessments for Sanfilippo syndrome.
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- Journal of Patient-Reported Outcomes, 2022, v. 6, n. 1, p. 1, doi. 10.1186/s41687-022-00447-w
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- Article
Targeting of the CNS in MPS-IH Using a Nonviral Transferrin-α-L-iduronidase Fusion Gene Product.
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- Molecular Therapy, 2008, v. 16, n. 8, p. 1459, doi. 10.1038/mt.2008.119
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- Article
Regression Trends & Treatment Effectiveness to Improve Quality of Life for a Pre-Adolescent Girl with MPS IIIA.
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- Journal of Developmental & Physical Disabilities, 2018, v. 30, n. 4, p. 545, doi. 10.1007/s10882-018-9601-5
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- Article
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.
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- 2019
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- Publication type:
- journal article
Correction to: 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.
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- 2019
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- Publication type:
- corrected article
Pharmacokinetic and Pharmacodynamic Evaluation of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients with Morquio A Syndrome.
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- Clinical Pharmacokinetics, 2014, v. 53, n. 12, p. 1137, doi. 10.1007/s40262-014-0173-y
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- Article
Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure.
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- 2011
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- Publication type:
- journal article
Transplantation of human umbilical mesenchymal stem cells cures the corneal defects of mucopolysaccharidosis VII mice.
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- Stem Cells, 2013, v. 31, n. 10, p. 2116, doi. 10.1002/stem.1481
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- Article
Mucopolysaccharidosis: A broad review.
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- 2022
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- Publication type:
- journal article
Anesthetic care and perioperative complications of children with Morquio syndrome.
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- Pediatric Anesthesia, 2012, v. 22, n. 9, p. 901, doi. 10.1111/j.1460-9592.2012.03904.x
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- Article
Does enzyme replacement therapy influence the ocular changes in type VI mucopolysaccharidosis?
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- Graefe's Archive of Clinical & Experimental Ophthalmology, 2009, v. 247, n. 7, p. 975, doi. 10.1007/s00417-008-1030-1
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- Article
Anterior Hypopituitarism and Treatment Response in Hunter Syndrome: A Comparison of Two Patients.
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- 2016
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- Publication type:
- Case Study
Ocular manifestations and management recommendations of lysosomal storage disorders I: mucopolysaccharidoses.
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- Clinical Ophthalmology, 2015, v. 9, p. 1633, doi. 10.2147/OPTH.S78368
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- Article
Miscellaneous.
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- Current Medical Literature: Growth, Growth Hormone, & Metabolism, 2010, v. 3, n. 1, p. 19
- Publication type:
- Article
Sanfilippo syndrome: consensus guidelines for clinical care.
- Published in:
- 2022
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- Publication type:
- journal article