Research Topics
| Patricia DicksonSummaryAffiliation: University of California Country: USA Publications
Research Grants
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Detail Information
Publications
Costello syndrome with pancreatic islet cell hyperplasiaPatricia I Dickson
Division of Medical Genetics, Harbor UCLA Medical Center, 1124 W Carson Street, Torrance, CA 90502, USA
Am J Med Genet A 130:402-5. 2004..Clinical similarities among these disorders and CS, together with the finding of islet cell hyperplasia, suggest that they may be related...
Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluidPatricia Dickson
Division of Medical Genetics, Department of Pediatrics, LA Biomed at Harbor UCLA, Torrance, CA 90502, USA
Mol Genet Metab 91:61-8. 2007..The improvement in GAG storage levels persisted three months after the final IT dose. The successful use of enzyme therapy via the CSF represents a potentially useful approach for lysosomal storage disorders...
Intrathecal enzyme replacement therapy for mucopolysaccharidosis I: translating success in animal models to patientsPatricia I Dickson
Los Angeles Biomedical Research Institute at Harbor UCLA Medical Center, Torrance, CA 90502, USA
Curr Pharm Biotechnol 12:946-55. 2011....
Research challenges in central nervous system manifestations of inborn errors of metabolismP I Dickson
Department of Pediatrics, LA Biomedical Research Institute at Harbor UCLA, 1124 W Carson St, HH1, Torrance, CA 90502, USA
Mol Genet Metab 102:326-38. 2011..Scientific, ethical and regulatory issues are discussed, along with ways to measure outcomes and the conduct of clinical trials. Participants included regulatory and funding agencies, clinicians, scientists, industry and advocacy groups...
Novel treatments and future perspectives: outcomes of intrathecal drug deliveryP I Dickson
Division of Medical Genetics, Department of Pediatrics, Los Angeles Biomedical Research Institute at Harbor UCLA Medical Center, Torrance, CA 90502, USA
Int J Clin Pharmacol Ther 47:S124-7. 2009..Studies are underway to investigate the safety and efficacy of intrathecal ERT in patients with MPS I...
Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type IPatricia I Dickson
Department of Pediatrics, Los Angeles Biomedical Research Institute at Harbor UCLA Medical Center, 1124 W Carson Street, Torrance, CA 90502, USA
Mol Genet Metab 101:115-22. 2010..High-dose IV rhIDU from birth (1.57mg/kg weekly) appeared to prevent cord compression due to protrusion of spinal disks...
Glycosaminoglycan storage in neuroanatomical regions of mucopolysaccharidosis I dogs following intrathecal recombinant human iduronidaseAgnes Chen
Division of Pediatric Neurology, Department of Pediatrics, LABioMed at Harbor UCLA Medical Center, Torrance, CA 90509, USA
APMIS 119:513-21. 2011..Response to therapy appeared to be greater in more superficial regions of the brain, particularly in the frontal lobe cortex...
Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis IPatricia Dickson
Division of Medical Genetics, Los Angeles Biomedical Research Institute at Harbor UCLA Medical Center, Torrance, California, USA
J Clin Invest 118:2868-76. 2008....
Hereditary multiple exostoses with spine involvement in a 4-year-old boyNavid Ezra
David Geffen School of Medicine at UCLA, Los Angeles, CA 90509, USA
Am J Med Genet A 152:1264-7. 2010..His affected sibling also displayed spinal cord compression because of a bony exostosis. Acute cervical spinal cord compression resulting from osteochondroma is a serious complication of HME. (c) 2010 Wiley-Liss, Inc...
Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compressionMaría Verónica Muñoz-Rojas
Hospital de Clinicas de Porto Alegre, Medical Genetics Service, Porto Alegre, Brazil
Am J Med Genet A 146:2538-44. 2008..6% of predicted %. In conclusion, although the improvement observed in this case with IT laronidase should be confirmed in further patients, this procedure seems to be a safe treatment for SCC in MPS I...
Research Grants
- Intrathecal enzyme therapy for mucopolysaccharidosis IPatricia Dickson; Fiscal Year: 2007..This research investigates intrathecal enzyme therapy for canine MRS I with translation to the first clinical trial of intrathecal enzyme replacement therapy for MRS I patients. ..
- Pilot/Phase I Study of Intrathecal Laronidase for Spinal Cord Compression in MucoPatricia Dickson; Fiscal Year: 2009..If successful, delivery into the spinal fluid could represent a practical, straightforward method of treating central nervous system disease due to lysosomal storage. ..
- Intrathecal enzyme therapy for mucopolysaccharidosis IPatricia I Dickson; Fiscal Year: 2010..This research investigates intrathecal enzyme therapy for canine MRS I with translation to the first clinical trial of intrathecal enzyme replacement therapy for MRS I patients. ..
