Status | Study |
Recruiting |
Study Name: Biomarker for Maroteaux-Lamy Disease Condition: Maroteaux-lamy Disease Lysosomal Storage Diseases Date: 2011-10-24 |
Recruiting |
Study Name: Biomarker for Pompe Disease Condition: Lysosomal Storage Diseases Pompe Disease Date: 2011-10-21 |
Recruiting |
Study Name: Biomarker for Morquio Disease Condition: Lysosomal Storage Diseases Morquio Disease Date: 2011-10-21 |
Recruiting |
Study Name: Biomarker for Krabbe Disease Condition: Lysosomal Storage Diseases Krabbe Disease Date: 2011-08-29 |
Active, not recruiting |
Study Name: Characterization of the Patient Population With Galactosialidosis Condition: Galactosialidosis Date: 2011-08-11 |
Recruiting |
Study Name: Biomarker for Gaucher Disease Condition: Lysosomal Storage Diseases Gaucher Disease Date: 2011-04-06 |
Recruiting |
Study Name: Biomarker for Hunter Disease Condition: Lysosomal Storage Diseases Hunter Disease Date: 2011-04-04 |
Withdrawn |
Study Name: Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases Condition: Lysosomal Storage Diseases Inborn Errors of Metabolism Date: 2009-10-20 Interventions: Biological: ALD-601 ALD-601 is manufactured by Aldagen from the 20% compartment of the selected umbilica |
Completed |
Study Name: A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I Condition: Cognitive Decline Mucopolysaccharidosis I Hurler-Scheie Sy Date: 2009-02-25 Interventions: Drug: laronidase For the treatment group, intrathecal rhIDU injections will consist of 3 cc of Aldurazym |
Terminated |
Study Name: Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I Condition: Spinal Cord Compression Mucopolysaccharidosis I Hurler-Sch Date: 2008-06-17 Interventions: Drug: laronidase 1.74 mg intrathecally every 1-3 months for 1 year |