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NCT05619900
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +36 more
- Locations
-
- University of California San Francisco San Francisco, California, United States
Recruiting
MPS (RaDiCo Cohort) (RaDiCo-MPS)
NCT06036693
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +33 more
- Locations
-
- Centre Hospitalier Universitaire d'Angers Angers, France
- Hôpital des Enfants - Groupe Hospitalier Pellegrin Bordeaux, France
- Hôpital Morvan Brest, France
- Hôpital d'Estaing Clermont-Ferrand, France
NCT01962415
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +125 more
- Locations
-
- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
NCT02254863
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
- Locations
-
- Duke University Medical Center Durham, North Carolina, United States
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Ann and Robert H Lurie Children's Hospital of Chicago Chicago, Illinois, United States
- Hackensack University Medical Center Hackensack, New Jersey, United States
- UNC Children's Research Institute Chapel Hill, North Carolina, United States
NCT05208281
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Cognition Disorders, Cognitive Dysfunction, +30 more
- Locations
-
- Federal State-Funded Healthcare Institution Central Clinical Hospital of the Russian Academy of Sciences (Research Institute of Pediatrics and Child Health Protection of the Central Clinical Hospital of the Russian Academy of Sciences) Moscow, Russia
- Federal State Budgetary Educational Institution of Higher Education "St. Petersburg State Pediatric Medical University" of the Ministry of Health of the Russian Federation Saint Petersburg, Russia
- V.I. Vernadsky Crimean Federal University Simferopol, Russia
- State Budgetary Healthcare Institution Republican Medical Genetic Center Ufa, Russia
Enrolling by Invitation
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
NCT05368038
- Conditions
- Acid Sphingomyelinase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +65 more
- Locations
-
- Maimonides Medical Center Brooklyn, New York, United States
- NYU Langone Hospital - Brooklyn Brooklyn, New York, United States
- North Shore University Hospital Manhasset, New York, United States
- NYU Langone Health - Tisch Hospital New York, United States
Enrolling by Invitation
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
NCT06075537
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Ann and Robert H Lurie Children's Hospital of Chicago Chicago, Illinois, United States
- Hackensack University Medical Center Hackensack, New Jersey, United States
- UNC Children's Research Institute Chapel Hill, North Carolina, United States
Enrolling by Invitation
An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects
NCT05594992
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
- Hospital Universitario Austral Buenos Aires, Argentina
- Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil
NCT02455622
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +17 more
- Locations
-
- Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
- Hospital Infantil Dr Robert Reid Cabral Santo Domingo, Dominican Republic
- Universitätsmedizin der Johannes Gutenberg-Universität Mainz Mainz, Germany
- Hospital Kuala Lumpur Kuala Lumpur, Malaysia
Completed
An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta)
NCT07344376
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +27 more
- Locations
-
- Samsung Medical Center Seoul, South Korea
Completed
Baby Detect : Genomic Newborn Screening
NCT05687474
- Conditions
- 3-Hydroxy 3-Methyl Glutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency, +368 more
- Locations
-
- CRMN, Hôpital La Citadelle Liège, Wallonia, Belgium
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +23 more
- Locations
-
- Ann & Robert H Lurie Childrens Hospital of Chicago Chicago, Illinois, United States
- University of North Carolina at Chapel Hill Chapel Hill, North Carolina, United States
- Legacy Emanuel Hospital Portland, Oregon, United States
- Children's Hospital of Pittsburgh of UPMC Pittsburgh, Pennsylvania, United States
NCT03359213
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
- Locations
-
- Grupo de Pesquisa Clínica em Genética Médica - HCPA Porto Alegre, Brazil
- Igeim - Unifesp São Paulo, Brazil
NCT02412787
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +17 more
- Locations
-
- Children's Hospital and Research Center at Oakland Oakland, California, United States
- Ann & Robert H Lurie Children's Hospital of Chicago Chicago, Illinois, United States
- University of North Carolina Chapel Hill, North Carolina, United States
- Women's and Children's Hospital Adelaide, Australia
NCT04571970
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +23 more
- Locations
-
- University of California San Francisco, Benioff Children's Hospital Oakland, California, United States
- McGill University Heath Center Montreal, Quebec, Canada
Completed
A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India
NCT05058391
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +17 more
- Locations
-
- SAT Hospital - Govt Medical College Thiruvananthapuram, Kerala, India
- JK Lone Hospital Jaipur, Rajasthan, India
- Institute of Child Health Kolkata, India
- All India Institute of Medical Sciences (AIIMS) New Delhi, India
Completed
Hunter Outcome Survey (HOS)
NCT03292887
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +17 more
- Locations
-
- Shire Lexington, Massachusetts, United States
NCT06475404
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +19 more
- Locations
-
- State budgetary healthcare institution of the city of Moscow "City Clinic No. 2 of the Moscow Health Department" Moscow, Russia
Completed
A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
NCT04007536
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland, California, United States
- UNC Children's Research Institute Chapel Hill, North Carolina, United States
- UPMC | Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
- Erasmus Medical Center Rotterdam, South Holland, Netherlands