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.

Giugliani, Roberto

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