Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study. [electronic resource]
- American journal of medical genetics. Part A Aug 2014
- 1953-64 p. digital
Publication Type: Journal Article; Multicenter Study; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
1552-4833
10.1002/ajmg.a.36584 doi
Adolescent Body Weights and Measures Child Child, Preschool Cross-Sectional Studies Enzyme Replacement Therapy Exercise Test Female Follow-Up Studies Heart Function Tests Humans Male Mucopolysaccharidosis VI--drug therapy N-Acetylgalactosamine-4-Sulfatase--therapeutic use Quality of Life Recombinant Proteins--therapeutic use Respiratory Function Tests Young Adult