39 studies found for:    Open Studies | "Mucopolysaccharidoses"
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Rank Status Study
21 Recruiting Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)
Conditions: Morquio A Syndrome;   Mucopolysaccharidosis IV A
Intervention:
22 Recruiting Natural History Study to Characterise the Course of Disease Progression in Patients With Mucopolysaccharidosis Type IIIB
Condition: MPS IIIB (Sanfilippo B Syndrome)
Intervention:
23 Recruiting A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
Condition: MPS IIIB (Sanfilippo Syndrome)
Intervention:
24 Recruiting The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients
Condition: Hunter Syndrome
Intervention:
25 Unknown  Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.
Conditions: Mucopolysaccharidosis;   Fabry Disease
Intervention:
26 Recruiting Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products
Conditions: Severe Combined Immunodeficiency;   Malignancy, Hematologic;   Neuroblastoma;   Neoplasm;   Mucopolysaccharidosis I
Intervention: Procedure: Venipuncture
27 Recruiting UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Conditions: Adrenoleukodystrophy;   Batten Disease;   Mucopolysaccharidosis II;   Leukodystrophy, Globoid Cell;   Leukodystrophy, Metachromatic;   Neimann Pick Disease;   Pelizaeus-Merzbacher Disease;   Sandhoff Disease;   Tay-Sachs Disease;   Brain Diseases, Metabolic, Inborn
Intervention: Biological: DUOC-01
28 Recruiting Biomarker for Sly Disease
Conditions: Mucopolysaccharidosis VII;   Sly Disease;   Beta-Glucuronidase Deficiency;   GUSB Deficiency
Intervention:
29 Recruiting Biomarker for Hurler Disease
Conditions: Mucopolysaccharidosis Type I;   MPS I;   Hurler-Scheie Syndrome;   Gargoylism, Hurler Syndrome
Intervention:
30 Recruiting Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
Conditions: Mucopolysaccharidosis Type IH;   MPS I;   Hurler Syndrome
Intervention: Drug: Laronidase
31 Recruiting A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Conditions: Mucopolysaccharidosis IV Type A;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: Vimizim® (elosulfase alfa)
32 Recruiting Biomarker for Maroteaux-Lamy Disease
Conditions: Maroteaux-lamy Disease;   Lysosomal Storage Diseases
Intervention:
33 Recruiting Biomarker for Morquio Disease
Conditions: Lysosomal Storage Diseases;   Morquio Disease
Intervention:
34 Not yet recruiting Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age
Condition: Hunter Syndrome
Intervention: Drug: Elaprase for intravenous (IV) infusion
35 Not yet recruiting Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
Condition: Maroteaux-Lamy Syndrome
Intervention: Drug: Naglazyme®
36 Recruiting Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment
Condition: Hunter Syndrome
Interventions: Biological: idursulfase-IT;   Other: No IT treatment
37 Recruiting Gait Analysis in MPS IVA
Conditions: MPS IVA;   Morquio Syndrome
Intervention:
38 Recruiting Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
Conditions: Primary Immune Deficiency Disorders;   Hemophagocytic Lymphohistiocytosis;   Inherited Bone Marrow Failure Syndrome;   Hemoglobinopathies;   Metabolic Disorders
Intervention: Biological: BPX-501 and AP1903
39 Recruiting Reduced Intensity Conditioning in Patients Aged ≤35 With Non-Malignant Disorders Undergoing UCBT, BMT, or PBSCT
Conditions: Primary Immunodeficiency Syndromes;   Congenital Bone Marrow Failure Syndromes;   Inherited Metabolic Disorders (IMD);   Hereditary Anemias;   Patients With Sickle Disease Presenting Specific Symptoms
Interventions: Drug: Hydroxyurea;   Drug: Alemtuzumab;   Drug: Fludarabine;   Drug: Melphalan;   Drug: Thiotepa

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Indicates status has not been verified in more than two years