Gene Therapy in Patients With Mucopolysaccharidosis Disease

Mucopolysaccharidosis type VI

Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project) A Study in MPS VI to Assess Safety and Efficacy of Odiparcil Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI Stem Cell Transplantation for Hurler Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network Clinical Trial of Growth Hormone in MPS I, II, and VI Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI Gene Therapy in Patients With Mucopolysaccharidosis Disease Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02 Study of rhASB in Patients With Mucopolysaccharidosis VI A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome