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Found 28 studies with search of: Open Studies | "Mucopolysaccharidoses"
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Rank Status Study
1 Recruiting Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis
Condition: Mucopolysaccharidosis
Interventions: Drug: Conditioning regimen;   Drug: Graft-versus-host disease (GVHD) prophylaxis;   Procedure: Stem Cell Transplantation
2 Recruiting Mucopolysaccharidosis I (MPS I) Registry
Condition: Mucopolysaccharidosis I
Intervention:
3 Not yet recruiting Human Placental-Derived Stem Cell Transplantation
Conditions: Mucopolysaccharidosis I;   Mucopolysaccharidosis VI;   Adrenoleukodystrophy;   Niemann-Pick Disease;   Metachromatic Leukodystrophy;   Wolman Disease;   Krabbe's Disease;   Gaucher's Disease;   Fucosidosis;   Batten Disease;   Severe Aplastic Anemia;   Diamond-Blackfan Anemia;   Amegakaryocytic Thrombocytopenia;   Myelodysplastic Syndrome;   Acute Myelogenous Leukemia;   Acute Lymphocytic Leukemia
Intervention: Drug: Human Placental Derived Stem Cell
4 Recruiting A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
Conditions: Mucopolysaccharidosis I;   Hurler's Syndrome;   Hurler-Scheie Syndrome;   Scheie
Intervention: Biological: Aldurazyme (laronidase)
5 Recruiting Longitudinal Study of Bone Disease in Children With Mucopolysaccharidoses (MPS) I, II, and VI
Condition: Mucopolysaccharidoses
Intervention:
6 Recruiting A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
Conditions: Cognitive Decline;   Mucopolysaccharidosis I;   Hurler-Scheie Syndrome;   Scheie Syndrome;   Lysosomal Storage Disease
Intervention: Drug: laronidase
7 Recruiting Biomarker for Hunter Disease
Conditions: Lysosomal Storage Diseases;   Hunter Disease;   Mucopolysaccharidoses
Intervention:
8 Recruiting Immune Tolerance Study With Aldurazyme® (Laronidase)
Condition: Mucopolysaccharidosis I
Interventions: Biological: laronidase;   Drug: Neoral® (CsA);   Drug: Imuran® (Aza)
9 Recruiting Carotid Structure and Function in MPS Syndromes
Conditions: MPS I;   MPS II;   MPS VI;   Mucopolysaccharidoses
Intervention:
10 Recruiting Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
Condition: Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)
Intervention:
11 Recruiting A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
Condition: MPS IV A
Intervention:
12 Recruiting Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Conditions: Mucopolysaccharidosis;   Hurler Syndrome;   Hunter Syndrome;   Maroteaux-Lamy Syndrome;   Sly Syndrome;   Alpha Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Adrenoleukodystrophy (ALD);   Globoid Cell Leukodystrophy (GLD);   Krabbe Disease;   Metachromatic Leukodystrophy (MLD);   Sphingolipidoses;   Peroxisomal Disorders
Interventions: Drug: Campath-1H;   Drug: Cyclophosphamide;   Drug: Busulfan;   Procedure: Allogeneic stem cell transplantation
13 Not yet recruiting A Study to Test the Possibility of Cross Reaction Induced by the ELAPRASE (R) Drug to GSK2788723 ELAPRASE is a Trade Mark Owned by a Third Party
Condition: Mucopolysaccharidosis II
Interventions: Drug: ELAPRASE (R);   Drug: GSK2788723
14 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
15 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
16 Recruiting Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
Conditions: Mucopolysaccharidosis Type III A;   Sanfilippo Disease Type A
Intervention: Genetic: SAF-301
17 Recruiting Natural History Study of Patients With Mucopolysaccharidosis Type III (MPS IIIB, Sanfilippo Syndrome Type B)
Condition: Sanfilippo Syndrome Type B
Intervention:
18 Recruiting Intrathecal Enzyme Replacement for Hurler Syndrome
Condition: Hurler Syndrome
Intervention: Drug: IRT Laronidase
19 Recruiting Biomarker for Maroteaux-Lamy Disease
Conditions: Maroteaux-lamy Disease;   Lysosomal Storage Diseases
Intervention:
20 Recruiting Biomarker for Morquio Disease
Conditions: Lysosomal Storage Diseases;   Morquio Disease
Intervention:

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