| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 8390756 | Molecular Genetics and Metabolism Reports | 2016 | 4 Pages |
Abstract
Conclusion: early ERT treatment did not improve the bone outcome in this severe MPS IV A patient after the 30Â months-long treatment. A longer term follow up is required to further assess the efficacy of ERT on both the motor and the respiratory function of the patient.
Related Topics
Life Sciences
Biochemistry, Genetics and Molecular Biology
Biochemistry, Genetics and Molecular Biology (General)
Authors
J. Do Cao, A. Wiedemann, T. Quinaux, S.F. Battaglia-Hsu, L. Mainard, R. Froissart, C. Bonnemains, S. Ragot, B. Leheup, P. Journeau, F. Feillet,