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Data from subjects receiving intrathecal laronidase for cervical spinal stenosis due to mucopolysaccharidosis type I.
Dickson, P I; Kaitila, I; Harmatz, P; Mlikotic, A; Chen, A H; Victoroff, A; Passage, M B; Madden, J; Le, S Q; Naylor, D E.
Afiliación
  • Dickson PI; Department of Pediatrics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, USA.
  • Kaitila I; Medical Genetics, University of Helsinki and Helsinki University Hospital, Helsinki, Finland.
  • Harmatz P; UCSF Benioff Children׳s Hospital Oakland, Oakland, CA, USA.
  • Mlikotic A; Department of Radiology, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, USA.
  • Chen AH; Department of Pediatrics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, USA ; Department of Neurology, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, USA.
  • Victoroff A; Department of Pediatrics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, USA.
  • Passage MB; Department of Pediatrics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, USA.
  • Madden J; UCSF Benioff Children׳s Hospital Oakland, Oakland, CA, USA.
  • Le SQ; Department of Pediatrics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, USA.
  • Naylor DE; Department of Neurology, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, USA.
Data Brief ; 5: 71-6, 2015 Dec.
Article en En | MEDLINE | ID: mdl-26484358
ABSTRACT
Five subjects with mucopolysaccharidosis type I and symptomatic cervical spinal stenosis received intrathecal laronidase in a 4-month pilot study and/or a 12-month extension study [1]. Clinical descriptions of study subjects, nonserious adverse events, individual data tables, and scoring system methods are provided. There were ten nonserious adverse events that occurred in more than one study subject. Somatosensory evoked potentials were absent in two subjects and normal in two subjects, limiting their utility as an endpoint. There were no significant changes in magnetic resonance imaging of cervical spinal cord or brain, pulmonary function tests, or cerebrospinal fluid opening pressure. These data are presented along with the scoring methods used in evaluation of the study subjects.
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Texto completo: 1 Bases de datos: MEDLINE Idioma: En Revista: Data Brief Año: 2015 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Bases de datos: MEDLINE Idioma: En Revista: Data Brief Año: 2015 Tipo del documento: Article País de afiliación: Estados Unidos