Esempio: Heart Attack

Prove cliniche su Mucopolisaccaridosi VII

Totale 13 risultati

NCT03604835

Reclutamento

Mucopolysaccharidosis VII Disease Monitoring Program

Condizioni: Mucopolysaccharidosis VII; MPS VII; MPS 7; Sly Syndrome
NCT02432144

Completato

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

Condizioni: Sly Syndrome; MPS VII; Mucopolysaccharidosis; Mucopolysaccharidosis VII
NCT02418455

Completato

Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

Condizioni: Sly Syndrome; MPS VII; Mucopolysaccharidosis; Mucopolysaccharidosis VII
NCT02230566

Completato

A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Condizioni: MPS 7; Sly Syndrome; Mucopolysaccharidosis; MPS VII
NCT03775174

Available

Expanded Access to Mepsevii

Condizioni: MPS VII; Mucopolysaccharidosis VII; Sly Syndrome
NCT02298699

Attivo, non reclutamento

Biomarker for Sly Disease (MPS VII) (BioSly)

Condizioni: Developmental Delay; Skeletal Abnormalities; Hepatomegaly; Splenomegaly
NCT01856218

Completato

An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Condizioni: Mucopolysaccharidosis Type 7
NCT02097251

No longer available

An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient

Condizioni: Mucopolysaccharidosis Type 7
NCT01870375

Completato

Longitudinal Studies of Brain Structure and Function in MPS Disorders

Condizioni: Mucopolysaccharidosis Type I; Mucopolysaccharidosis Type II; Mucopolysaccharidosis Type VI; Mucopolysaccharidosis Type IV; Mucopolysaccharidosis Type VII
NCT02171104

Reclutamento

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

Condizioni: Mucopolysaccharidosis Disorders; Hurler Syndrome; Hunter Syndrome; Maroteaux Lamy Syndrome; Sly Syndrome; Alpha-Mannosidosis; Fucosidosis; Aspartylglucosaminuria; Glycoprotein Metabolic Disorders; Sphingolipidoses; Recessive Leukodystrophies; Globoid Cell Leukodystrophy; Metachromatic Leukodystrophy; Niemann-Pick B; Niemann-Pick C Subtype 2; Sphingomyelin Deficiency; Peroxisomal Disorders; Adrenoleukodystrophy With Cerebral Involvement; Zellweger Syndrome; Neonatal Adrenoleukodystrophy; Infantile Refsum Disease; Acyl-CoA Oxidase Deficiency; D-Bifunctional Enzyme Deficiency; Multifunctional Enzyme Deficiency; Alpha-methylacyl-CoA Racmase Deficiency; Mitochondrial Neurogastrointestingal Encephalopathy; Severe Osteopetrosis; Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation); Inherited Metabolic Disorders
NCT00668564

Terminato

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

Condizioni: Hurler's Syndrome; Maroteaux-Lamy Syndrome; Sly Syndrome; Alpha Mannosidosis; Fucosidosis; Aspartylglucosaminuria; Sphingolipidoses; Krabbe Disease; Wolman's Disease; Niemann-Pick Disease Type B; Niemann-Pick Disease, Type C
NCT01043640

Completato

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Condizioni: Mucopolysaccharidosis; Hurler Syndrome; Hunter Syndrome; Maroteaux-Lamy Syndrome; Sly Syndrome; Alpha Mannosidosis; Fucosidosis; Aspartylglucosaminuria; Adrenoleukodystrophy (ALD); Krabbe Disease; Metachromatic Leukodystrophy (MLD); Sphingolipidoses; Peroxisomal Disorders
NCT04532047

Non ancora reclutando

In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

Condizioni: MPS I; MPS II; MPS IVA; MPS VI; Mps VII; Gaucher Disease, Type 2; Gaucher Disease, Type 3; Pompe Disease Infantile-Onset; Wolman Disease