| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 10833109 | Molecular Genetics and Metabolism | 2013 | 8 Pages |
Abstract
⺠MorCAP is the first natural history study with quantitative assessments of MPS IVA. ⺠Endurance, mobility, respiratory function, stature and quality of life are impacted. ⺠All affected individuals experience significant functional limitations. ⺠Increasing severity with age illustrates the progressive nature of Morquio A.
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Authors
Paul Harmatz, Karl Eugen Mengel, Roberto Giugliani, Vassili Valayannopoulos, Shuan-Pei Lin, Rossella Parini, Nathalie Guffon, Barbara K. Burton, Christian J. Hendriksz, John Mitchell, Ana Martins, Simon Jones, Norberto Guelbert, Ashok Vellodi,