447 studies found for:    "Lysosomal Storage Diseases" OR "Schindler disease"
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Rank Status Study
21 Terminated Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
Conditions: Mucopolysaccharidosis I;   Lysosomal Storage Diseases;   Spinal Cord Compression
Intervention: Drug: laronidase
22 Terminated ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Conditions: Inherited Metabolic Diseases;   Lysosomal Storage Disorders;   Peroxisomal Storage Diseases;   Inborn Errors of Metabolism;   Mucopolysaccharidosis
Intervention: Biological: ALD-101
23 Recruiting Characterization of the Patient Population With Galactosialidosis
Condition: Galactosialidosis
Intervention:
24 Recruiting Physician Initiated Request for Migalastat in Individual Patients With Fabry Disease
Condition: Fabry Disease
Intervention: Drug: migalastat HCl
25 Recruiting Nervous System Degeneration in Glycosphingolipid Storage Disorders
Conditions: Gangliosidoses;   Gaucher Disease
Intervention:
26 Recruiting Open-Label Phase 3 Long-Term Safety Study of Migalastat
Condition: Fabry Disease
Intervention: Drug: migalastat HCl 150mg
27 Completed Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
Condition: Gaucher Disease
Intervention: Procedure: Blood sample
28 Not yet recruiting Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
Condition: Fabry Disease
Intervention: Drug: migalastat HCl 150 mg
29 Unknown  Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
Condition: Gangliosidoses GM2
Intervention: Drug: miglustat
30 Recruiting Biomarker for Niemann Pick Type C Disease
Conditions: Niemann-Pick Disease;   Niemann-Pick Disease, Type C
Intervention:
31 Recruiting The Natural History of Mucolipidosis Type IV
Condition: Mucolipidosis Type IV
Intervention:
32 Recruiting Safety and Efficacy Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency
Condition: Lysosomal Acid Lipase Deficiency
Intervention: Drug: sebelipase alfa
33 Completed
Has Results
A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
Condition: Hunter Syndrome
Interventions: Other: Control;   Drug: Idursulfase IT (1 mg);   Drug: Idursulfase IT (10 mg);   Drug: Idursulfase IT (30 mg)
34 Completed
Has Results
Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase
Conditions: Hunter Syndrome;   Mucopolysaccharidosis II (MPS II)
Intervention: Biological: Idursulfase
35 Completed
Has Results
An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
Condition: Hunter Syndrome
Intervention: Biological: Idursulfase
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 Completed
Has Results
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
Conditions: Hunter Syndrome;   Mucopolysaccharidosis II;   MPS II
Intervention: Biological: Idursulfase
38 Active, not recruiting Extension of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase (IT)in Conjunction With Elaprase in Pediatric Patients With Hunter Syndrome and Cognitive Impairment
Condition: Hunter Syndrome
Intervention: Drug: Idursulfase-IT
39 Completed
Has Results
Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase
Condition: Gaucher Disease
Intervention: Drug: Taliglucerase alfa
40 Recruiting Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations
Conditions: Glucocerebrosidase Mutations;   Gaucher Disease
Intervention:

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Indicates status has not been verified in more than two years