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Results for “Aspartylglucosaminuria”

7 trials

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Showing 7 of 7 results

Testing effectiveness (Phase 2)Ended earlyNCT00668564
What this trial is testing

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Who this might be right for
Hurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more
Masonic Cancer Center, University of Minnesota 18
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104
What this trial is testing

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Who this might be right for
Mucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more
Masonic Cancer Center, University of Minnesota 149
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07530796
What this trial is testing

Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU)

Who this might be right for
AspartylglucosaminuriaAspartylglucosamidase (AGA) Deficiency
Rare Trait Hope 9
Not applicableStudy completedNCT01891422
What this trial is testing

Longitudinal Studies of the Glycoproteinoses

Who this might be right for
AspartylglucosaminuriaFucosidosisGalactosialidosis+6 more
Greenwood Genetic Center 100
Not applicableEnded earlyNCT03853876
What this trial is testing

A Natural History Study of Aspartylglucosaminuria

Who this might be right for
AspartylglucosaminuriaAspartylglucosamidase (AGA) DeficiencyLysosomal Storage Diseases
Neurogene Inc. 8
Testing effectiveness (Phase 2)Study completedNCT01043640
What this trial is testing

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Who this might be right for
MucopolysaccharidosisHurler SyndromeHunter Syndrome+10 more
Masonic Cancer Center, University of Minnesota 46