| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 10832538 | Molecular Genetics and Metabolism | 2016 | 12 Pages |
Abstract
In conclusion, we have shown the utility of the proposed ADL questionnaire and frequency of surgical interventions in patients with MPS IVA to evaluate the clinical severity and therapeutic efficacy compared with age-matched controls.
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Authors
Eriko Yasuda, Yasuyuki Suzuki, Tsutomu Shimada, Kazuki Sawamoto, William G. Mackenzie, Mary C. Theroux, Christian Pizarro, Li Xie, Freeman Miller, Tariq Rahman, Heidi H. Kecskemethy, Kyoko Nagao, Thierry Morlet, Thomas H. Shaffer, Yasutsugu Chinen,