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NCT00029965
- Conditions
- Bone Diseases, Bone Diseases, Metabolic, Brain Atrophy, +29 more
- Locations
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- National Institutes of Health Clinical Center Bethesda, Maryland, 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
The Natural History of Sialidosis Type I
NCT06316752
- Conditions
- Bone Diseases, Bone Diseases, Metabolic, Brain Diseases, +15 more
- Locations
-
- National Taiwan University Hospital Taipei, Taiwan
Enrolling by Invitation
Safety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IV
NCT07398872
- Conditions
- Bone Diseases, Bone Diseases, Metabolic, Brain Diseases, +15 more
- Locations
-
- Children's Hospital Zhejiang University School of Medicine Hangzhou, Zhejiang, China
NCT01891422
- Conditions
- Alpha Mannosidosis, Aspartylglucosaminuria, Beta Mannosidosis, +29 more
- Locations
-
- Greenwood Genetic Center North Charleston, South Carolina, United States
NCT00383448
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
- Locations
-
- Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States
Completed
Stem Cell Transplantation for Hurler
NCT00176917
- Conditions
- Bone Diseases, Bone Diseases, Metabolic, Brain Diseases, +23 more
- Locations
-
- Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States
NCT01626092
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +50 more
- Locations
-
- Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States
NCT00015782
- Conditions
- Bone Diseases, Bone Diseases, Metabolic, Brain Diseases, +17 more
- Locations
-
- National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, Maryland, United States
Active, Not Recruiting
Mucolipidosis Type IV Natural History Study
NCT05782387
- Conditions
- Bone Diseases, Bone Diseases, Metabolic, Brain Diseases, +15 more
- Locations
-
- Massachusetts General Hospital Boston, Massachusetts, United States