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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Not applicableStudy completedNCT01035424
What this trial is testing

Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis

Who this might be right for
Batten DiseaseLate Infantile Neuronal Ceroid Lipofuscinosis
Weill Medical College of Cornell University 48
Testing effectiveness (Phase 2)Study completedNCT02725580
What this trial is testing

Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease

Who this might be right for
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
Emily de los Reyes 13
Not applicableStudy completedNCT05923788
What this trial is testing

T1 Mapping in Fabry Disease

Who this might be right for
Fabry Disease
Hospices Civils de Lyon 70
Early research (Phase 1)Study completedNCT02536911
What this trial is testing

The Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate

Who this might be right for
Gaucher Disease
Genzyme, a Sanofi Company 24
Not applicableLooking for participantsNCT04440254
What this trial is testing

Long Duration Holter ECG in Fabry Disease

Who this might be right for
Fabry Disease
Institut National de la Santé Et de la Recherche Médicale, France 40
Large-scale testing (Phase 3)Study completedNCT00705939
What this trial is testing

Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Who this might be right for
Gaucher Disease
Pfizer 45
Not applicableLooking for participantsNCT03333200
What this trial is testing

Longitudinal Study of Neurodegenerative Disorders

Who this might be right for
MLDKrabbe DiseaseALD+24 more
University of Pittsburgh 1,500
Not applicableStudy completedNCT02785744
What this trial is testing

Genzyme Osteopenia/Osteoporosis Study

Who this might be right for
Gaucher Disease
NYU Langone Health 76
Not applicableActive Not RecruitingNCT07011329
What this trial is testing

Brain Alterations Based on Multiplex MR Imaging in Nasopharyngeal Carcinoma Patients After Treatment, and Their Correlation With Clinical Cognitive Scores.

Who this might be right for
Nasopharyngeal Carcinoma (NPC)
Hubei Cancer Hospital 60
Not applicableWithdrawnNCT02298647
What this trial is testing

Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2)

Who this might be right for
Hepato-splenomegalyDysostosis MultiplexSeizures+1 more
CENTOGENE GmbH Rostock
Not applicableStudy completedNCT02798458
What this trial is testing

Evaluation of the Gastrointestinal Manifestation of Fabry's Disease

Who this might be right for
Fabry's Disease
Massachusetts General Hospital 48
Large-scale testing (Phase 3)Study completedNCT01760564
What this trial is testing

Application of Miglustat in Patients With Niemann-Pick Type C

Who this might be right for
Niemann-Pick Disease Type C
National Taiwan University Hospital 5
Not applicableActive Not RecruitingNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Not applicableStudy completedNCT04353466
What this trial is testing

Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs

Who this might be right for
Gaucher Disease, Type 1
Shaare Zedek Medical Center 30
Not applicableStudy completedNCT02023086
What this trial is testing

Clinical Investigation on the Blood Oxygenation at the Optic Nerve Head in Fabry Patients

Who this might be right for
Fabry Disease
Université de Montréal 8
Not applicableNot Yet RecruitingNCT07485660
What this trial is testing

Care Pathway for Patients With Fabry's Disease (Fabry-PATH)

Who this might be right for
Fabry Disease
University Hospital, Toulouse 200
Not applicableWithdrawnNCT01331642
What this trial is testing

Biomarker for Gaucher Disease: BioGaucher (BioGaucher)

Who this might be right for
SplenomegalyHepatomegaly
CENTOGENE GmbH Rostock
Not applicableStudy completedNCT07285369
What this trial is testing

High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)

Who this might be right for
Gaucher Disease, Type 3
Agyany Pharma LTD 12
Not applicableLooking for participantsNCT07758816
What this trial is testing

Advancing Integrated Therapies for Gaucher Disease

Who this might be right for
Gaucher Disease
Yale University 30
Not applicableEnded earlyNCT03321604
What this trial is testing

Kidney Information Network for Disease Research and Education

Who this might be right for
ESRDFabry DiseaseRare Diseases+3 more
Massachusetts General Hospital 338
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