Works matching DE "MUCOPOLYSACCHARIDOSIS treatment"
Results: 71
Abstract- Poster.
- Published in:
- 2022
- Publication type:
- Abstract
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
Thrombocytopenia associated with galsulfase treatment.
- Published in:
- 2011
- By:
- Publication type:
- Case Study
Expensive drugs for rare disorders: to treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI.
- Published in:
- Current Medical Research & Opinion, 2009, v. 25, n. 5, p. 1285, doi. 10.1185/03007990902892633
- By:
- Publication type:
- Article
Elosulfase alfa (BMN 110) for the treatment of mucopolysaccharidosis IVA (Morquio A Syndrome).
- Published in:
- Expert Review of Clinical Pharmacology, 2016, v. 9, n. 12, p. 1521, doi. 10.1080/17512433.2017.1260000
- By:
- Publication type:
- Article
Elosulfase alfa for the treatment of mucopolysacchoridosis IVA.
- Published in:
- Expert Review of Endocrinology & Metabolism, 2015, v. 10, n. 6, p. 569, doi. 10.1586/17446651.2015.1091723
- By:
- Publication type:
- Article
Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration.
- Published in:
- Journal of Molecular Medicine, 2017, v. 95, n. 10, p. 1043, doi. 10.1007/s00109-017-1562-0
- 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
Miscellaneous.
- Published in:
- Current Medical Literature: Growth, Growth Hormone, & Metabolism, 2010, v. 3, n. 1, p. 19
- 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
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
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
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
Anterior Hypopituitarism and Treatment Response in Hunter Syndrome: A Comparison of Two Patients.
- Published in:
- 2016
- By:
- Publication type:
- Case Study
Mucopolysaccharidosis VI: the Italian experience.
- Published in:
- 2009
- By:
- Publication type:
- journal article
A new case of mucopolysaccharidosis VII presenting as non immune hydrops fetalis.
- Published in:
- European Journal of Pediatrics, 2003, v. 162, n. 7/8, p. 520, doi. 10.1007/s00431-003-1198-7
- 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
Treatment of MPS I mice with microencapsulated cells overexpressing IDUA: effect of the prednisolone administration.
- Published in:
- Journal of Microencapsulation, 2013, v. 30, n. 4, p. 383, doi. 10.3109/02652048.2012.746745
- By:
- Publication type:
- Article
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 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
NDST1 Preferred Promoter Confirmation and Identification of Corresponding Transcriptional Inhibitors as Substrate Reduction Agents for Multiple Mucopolysaccharidosis Disorders.
- Published in:
- PLoS ONE, 2016, v. 11, n. 9, p. 1, doi. 10.1371/journal.pone.0162145
- By:
- Publication type:
- Article
Heart and Cardiovascular Involvement in Patients with Mucopolysaccharidosis Type IVA (Morquio-A Syndrome).
- Published in:
- PLoS ONE, 2016, v. 11, n. 9, p. 1, doi. 10.1371/journal.pone.0162612
- By:
- Publication type:
- Article
Update on mucopolysaccharidosis type II.
- Published in:
- Acta Paediatrica, 2007, p. 55, doi. 10.1111/j.1651-2227.2007.00210.x
- 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
Treatment of MPS VII (Sly disease) by allogeneic BMT in a female with homozygous A619V mutation.
- Published in:
- Bone Marrow Transplantation, 1998, v. 21, n. 6, p. 629, doi. 10.1038/sj.bmt.1701141
- By:
- Publication type:
- Article
Laronidase.
- Published in:
- BioDrugs, 2002, v. 16, n. 4, p. 316, doi. 10.2165/00063030-200216040-00009
- 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
Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy.
- 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
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
Anesthetic Management in Mucopolysaccharidoses.
- Published in:
- ISRN Anesthesiology, 2013, p. 1, doi. 10.1155/2013/791983
- By:
- Publication type:
- Article
Pentosan Polysulfate: Oral Versus Subcutaneous Injection in Mucopolysaccharidosis Type I Dogs.
- Published in:
- PLoS ONE, 2016, v. 11, n. 4, p. 1, doi. 10.1371/journal.pone.0153136
- 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
Activities of genes controlling sphingolipid metabolism in human fibroblasts treated with flavonoids.
- Published in:
- Metabolic Brain Disease, 2015, v. 30, n. 5, p. 1257, doi. 10.1007/s11011-015-9705-x
- 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
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
Anaesthetic implications of the changing management of patients with mucopolysaccharidosis.
- Published in:
- 2016
- By:
- Publication type:
- journal article
Drug Repurposing Patent Applications April-June 2015.
- Published in:
- Assay & Drug Development Technologies, 2015, v. 13, n. 10, p. 654, doi. 10.1089/adt.2015.29030.pq2
- 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
Mucopolysaccharidosis VI, a Case Report.
- Published in:
- Journal of Guilan University of Medical Sciences, 2014, v. 22, n. 89, p. 83
- By:
- Publication type:
- Article