433 studies found for:    "Lysosomal Storage Diseases" OR "Schindler disease"
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Rank Status Study
21 Recruiting Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
Conditions: Lysosomal Storage Disease;   Peroxisomal Disorder
Interventions: Drug: Campath-1H;   Drug: Clofarabine;   Drug: Melphalan;   Radiation: Total Body Irradiation with Marrow Boosting;   Biological: Hematopoietic stem cell transplantation;   Drug: Cyclosporine A;   Drug: Mycophenolate mofetil
22 Recruiting Physician Initiated Request for Migalastat in Individual Patients With Fabry Disease
Condition: Fabry Disease
Intervention: Drug: migalastat HCl
23 Recruiting Biomarker for Niemann Pick Type C Disease
Conditions: Niemann-Pick Disease;   Niemann-Pick Disease, Type C
Intervention:
24 Unknown  Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
Condition: Gangliosidoses GM2
Intervention: Drug: miglustat
25 Recruiting Characterization of the Patient Population With Galactosialidosis
Condition: Galactosialidosis
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 Safety and Efficacy Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency
Condition: Lysosomal Acid Lipase Deficiency
Intervention: Drug: sebelipase alfa
29 Recruiting The Natural History of Mucolipidosis Type IV
Condition: Mucolipidosis Type IV
Intervention:
30 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
31 Completed An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
Condition: Hunter Syndrome
Intervention: Biological: Idursulfase
32 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
33 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
34 Completed 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: Drug: Idursulfase-IT;   Other: Control
35 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
36 Completed Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
Condition: Fabry Disease
Interventions: Drug: migalastat hydrochloride;   Drug: Placebo
37 Recruiting A Study of the Effects of Fabrazyme (Agalsidase Beta) on Mother's Lactation and on the Growth, Development and Immunologic Response of Their Infants
Condition: Fabry Disease
Intervention: Biological: agalsidase beta
38 Recruiting A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers
Condition: Fabry Disease
Interventions: Drug: Migalastat HCl;   Drug: placebo
39 Recruiting Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
Condition: Neimann-Pick Disease
Intervention:
40 Enrolling by invitation Identification of Undiagnosed Gaucher Disease
Condition: Gaucher Disease
Intervention: Other: Enzyme analysis

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Indicates status has not been verified in more than two years