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NCT02780297
- Conditions
- Abnormalities, Multiple, Adenine Phosphoribosyltransferase Deficiency, Adenine phosphoribosyltransferase deficiency, +28 more
- Locations
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- University of Alabama @ Birmingham Birmingham, Alabama, United States
- Mayo Clinic Jacksonville Jacksonville, Florida, United States
- Children's Memorial Hospital Chicago, Illinois, United States
- Children's Hospital, Harvard Medical School Boston, Massachusetts, United States
Recruiting
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
NCT01793168
- Conditions
- 1p36 Deletion Syndrome, 3-Methylglutaconic Aciduria, Type V, 4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome, +698 more
- Locations
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- Sanford Health Sioux Falls, South Dakota, United States
- Online Patient Enrollment System Sydney, Australia
Recruiting
National Registry of Rare Kidney Diseases
NCT06065852
- Conditions
- AH Amyloidosis, AHL Amyloidosis, AL Amyloidosis, +207 more
- Locations
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- Zoe Plummer Bristol, South West, United Kingdom
Not Yet Recruiting
An Open-label, Phase 2 Pilot Study on the Efficacy and Safety of Piclidenoson in Patients With Lowe Syndrome
NCT07410455
- Conditions
- Abnormalities, Multiple, Amino Acid Transport Disorders, Inborn, Brain Diseases, +20 more
- Locations
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- IRCCS Ospedale Pediatrico Bambino Gesù Roma, Italy
NCT01314560
- Conditions
- Abnormalities, Multiple, Amino Acid Transport Disorders, Inborn, Brain Diseases, +25 more
- Locations
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- Necker Enfants Malades Hospital, Genetic Paris, France
NCT00359515
- Conditions
- Abnormalities, Multiple, Amino Acid Transport Disorders, Inborn, Brain Diseases, +23 more
- Locations
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- National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, Maryland, United States