Data from subjects receiving intrathecal laronidase for cervical spinal stenosis due to mucopolysaccharidosis type I
| Autor: | Alla Victoroff, Patricia I. Dickson, Paul Harmatz, Anton Mlikotic, Ilkka Kaitila, Merry Passage, Agnes Chen, Jacqueline Madden, Steven Q. Le, David E. Naylor |
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| Přispěvatelé: | Medicum, Department of Medical and Clinical Genetics |
| Jazyk: | angličtina |
| Rok vydání: | 2015 |
| Předmět: |
Pathology
medicine.medical_specialty education lcsh:Computer applications to medicine. Medical informatics Pulmonary function testing 03 medical and health sciences Mucopolysaccharidosis type I 0302 clinical medicine Cerebrospinal fluid medicine Adverse effect lcsh:Science (General) Data Article 0303 health sciences Multidisciplinary medicine.diagnostic_test business.industry 030302 biochemistry & molecular biology Cervical spinal stenosis Magnetic resonance imaging Spinal cord medicine.disease 3. Good health medicine.anatomical_structure Somatosensory evoked potential Anesthesia lcsh:R858-859.7 3111 Biomedicine business 030217 neurology & neurosurgery lcsh:Q1-390 |
| Zdroj: | Data in Brief, Vol 5, Iss C, Pp 71-76 (2015) Data in Brief |
| ISSN: | 2352-3409 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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