Clinical Trials on Acid Maltase Deficiency (AMD) in New Zealand
Total 1589 results
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Arcturus Therapeutics, Inc.CompletedOrnithine Transcarbamylase DeficiencyNew Zealand
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe DiseaseUnited States
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Shanghai Jiao Tong University School of MedicineShanghai Vitalgen BioPharma Co., Ltd.RecruitingAromatic L-amino Acid Decarboxylase (AADC) DeficiencyChina
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Swiss Federal Institute of TechnologyCompletedPregnancy Related | Iodine DeficiencyNew Zealand
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Genzyme, a Sanofi CompanyApproved for marketingGlycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe Disease (Late-Onset)United States
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AlexionRecruitingWolman Disease | Cholesterol Ester Storage Disease | Lysosomal Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 | Acid Lipase Deficiency | LIPA Deficiency | LAL-DeficiencyFrance, Belgium, United States, Spain, Germany, Greece, Israel, Italy, Slovenia, United Kingdom, Brazil, Canada, Denmark, Australia, Croatia, Czechia, Ireland, Mexico, Netherlands, Poland, Portugal, Saudi Arabia
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Genzyme, a Sanofi CompanyCompletedPompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2United States, France, Netherlands
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Arrowhead PharmaceuticalsCompleted
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Dicerna Pharmaceuticals, Inc.WithdrawnAlpha 1-Antitrypsin DeficiencyNew Zealand
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Genzyme, a Sanofi CompanyTerminatedGlycogenosis 2 | Glycogen Storage Disease Type II (GSD II) | Acid Maltase Deficiency | Pompe Disease (Infantile-Onset)United States, Taiwan, Germany
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyCompleted
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Pompe Disease | Glycogenesis 2 Acid Maltase DeficiencyUnited States, Canada, Australia
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Genzyme, a Sanofi CompanyCompletedGlycogenesis 2 Acid Maltase Deficiency | Pompe Disease (Late-Onset) | Glycogen Storage Disease Type II (GSD II)United States, Netherlands, United Kingdom, Germany
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Pompe Disease (Late-Onset) | Glycogenesis Type II | Acid Maltase Deficiency (AMD)United States, France, Canada, Netherlands, Australia
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CSL LimitedCompletedPrimary Immune DeficiencyAustralia, New Zealand
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AlexionCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited States, Canada, United Kingdom, Italy, France
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Genzyme, a Sanofi CompanyRecruitingPompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Glycogenesis 2 Acid Maltase DeficiencyCroatia, United States, Italy, Belgium, Czechia
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Baxter Healthcare CorporationCompletedEssential Fatty Acid Deficiency (EFAD)United States
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Institut PasteurUniversity of Copenhagen; Institut CochinCompletedDiarrhea | InfantileFrance, New Caledonia, Central African Republic
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Intellia TherapeuticsNot yet recruitingHereditary AngioedemaFrance, New Zealand, United Kingdom
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National Institute of Allergy and Infectious Diseases...National Center for Advancing Translational Sciences (NCATS); Primary Immune...Enrolling by invitationADA-SCID | Omenn Syndrome | SCID | Reticular Dysgenesis | XSCID | Leaky SCIDUnited States, Canada
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Mendel TuchmanChildren's Hospital of Philadelphia; University of California, Los Angeles; Icahn... and other collaboratorsCompletedMethylmalonic Acidemia | Carbamoyl-Phosphate Synthase I Deficiency Disease | Propionic Acidemia, Type I and/or Type II | Ornithine Carbamoyltransferase DeficiencyUnited States
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National Institute of Allergy and Infectious Diseases...National Center for Advancing Translational Sciences (NCATS); Office of Rare... and other collaboratorsEnrolling by invitationOmenn Syndrome | Reticular Dysgenesis | Severe Combined Immunodeficiency (SCID) | XSCID | Leaky SCID | ADA SCIDUnited States, Canada
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Baxalta now part of ShireCompletedAlpha 1-Antitrypsin DeficiencyNew Zealand, Australia
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Baxalta now part of ShireBaxter Healthcare, Ltd. (New Zealand), Baxter Healthcare Pty. Ltd. (Australia)CompletedAlpha 1-Antitrypsin DeficiencyAustralia, New Zealand
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Leadiant Biosciences, Inc.CompletedSevere Combined Immunodeficiency | ADA-SCID | Adenosine Deaminase DeficiencyUnited States
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD) | LAL-DeficiencyFrance, United States, United Kingdom, Czechia
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ViiV HealthcareCompletedHIV InfectionsUnited States, New Zealand
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Intellia TherapeuticsActive, not recruitingHereditary AngioedemaFrance, New Zealand, Germany, United Kingdom, Netherlands, Australia
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Ultragenyx Pharmaceutical IncActive, not recruitingOrnithine Transcarbamylase (OTC) DeficiencyCanada, United States, France, Spain, United Kingdom
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Arcturus Therapeutics, Inc.Active, not recruitingOrnithine Transcarbamylase DeficiencyUnited States
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Homology Medicines, IncEnrolling by invitationPhenylketonuria | PAH DeficiencyUnited States
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | LAL-Deficiency | Cholesterol Ester Storage Disease (CESD)France, United Kingdom, United States, Canada, Czechia
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Columbia UniversityCompletedAortocaval Compression | Fetal AcidosisUnited States
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New York Institute of TechnologyCompleted
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Jasper Therapeutics, Inc.Recruiting
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Alexion PharmaceuticalsTerminatedLysosomal Acid Lipase DeficiencyUnited States
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Hospital for Special Surgery, New YorkCompletedE-Aminocaproic Acid | Bilateral Varus Rotational OsteotomyUnited States
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Fu Jen Catholic UniversityCompletedVitamin B 12 DeficiencyTaiwan
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Murdoch Childrens Research InstituteRoyal Children's HospitalCompletedPrimary Immunodeficiency | Invasive Pneumococcal Disease, Protection Against | Invasive Pneumococcal Disease, Recurrent Isolated, 1 | Invasive Pneumococcal Disease, Recurrent Isolated, 2Australia, New Zealand
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Inhibrx, Inc.CompletedAlpha-1 Antitrypsin Deficiency | AATDUnited States, New Zealand, United Kingdom
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Hospital for Special Surgery, New YorkCompletedVitamin D DeficiencyUnited States
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Sitaram Bhartia Institute of Science and ResearchUniversity College, London; Public Health Foundation of India; Paropakar Matenity...CompletedVitamin B 12 DeficiencyIndia, Nepal
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CSL LimitedCompleted
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Italy, Canada, France, United Kingdom, Austria, Germany
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Ultragenyx Pharmaceutical IncRecruitingOTC DeficiencyUnited States, Portugal, Australia, Italy, Spain, United Kingdom, Japan, Canada, Germany, France, Argentina, Netherlands, Brazil
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Ascendis Pharma Endocrinology Division A/SCompletedA Safety, Tolerability and Efficacy Study of TransCon hGH in Children With Growth Hormone DeficiencyEndocrine System Diseases | Pituitary Diseases | Hormone Deficiency | Growth Hormone Deficiency, PediatricUnited States, Canada, Australia, New Zealand
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Icahn School of Medicine at Mount SinaiCompletedIron Deficiency AnemiaUnited States
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Montefiore Medical CenterGlenn Foundation for Medical ResearchWithdrawnFasting | Aging Well | Autophagy | Ketosis, MetabolicUnited States
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James J. Peters Veterans Affairs Medical CenterCompletedMacrocytic Anemia With Vitamin B12 DeficiencyUnited States