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Therapies of mucopolysaccharidosis IVA (Morquio A syndrome)

Shunji Tomatsu1, Carlos J Alméciga-Díaz2, Hector Barbosa2

  • 1Professor and Director, Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, USA ; Nemours/Alfred I. duPont Hospital for Children, Skeletal Dysplasia Center, Nemours Biomedical Research, 1600 Rockland Rd., Wilmington, DE 19803, USA.

Expert Opinion on Orphan Drugs
|November 25, 2014
PubMed
Abstract

Keywords:
GALNSenzyme replacement therapygene therapykeratan sulfatemucopolysaccharidosis IVAtandem mass spectrometry

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