Found 4 clinical trials
Venglustat in Combination With Cerezyme in Adult and Pediatric Patients With Gaucher Disease Type 3
Cohort 3 Evaluate the efficacy of venglustat in combination with Cerezyme in adult and pediatric GD3 patients by assessing: CSF Lyso-GL1 levels Modified Friedreichs Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Brain resting-state functional Magnetic Resonance Imaging (rs-fMRI) reflecting connectivity between parieto-occipital areas Bone disease manifestations Evaluate safety …
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- 04 Dec, 2020
- 7 locations
The Effectiveness of Megadose Shinbaro Pharmacopuncture for Patients With Chronic Shoulder Pain
This study is a prospective, case-control observational trial. The investigators will compare the Megadose Shinbaro Pharmacopuncture group to the control group to analyze the effectiveness of shoulder Megadose Shinbaro Pharmacopuncture.
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- 16 Feb, 2024
- 1 location
GROWing Up With Rare GENEtic Syndromes
Introduction Rare complex syndromes Patients with complex genetic syndromes, by definition, have combined medical problems affecting multiple organ systems, and intellectual disability is often part of the syndrome. During childhood, patients with rare genetic syndromes receive multidisciplinary and specialized medical care; they usually receive medical care from 3-4 medical specialists. …
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- 16 Feb, 2024
- 1 location
Bone-biomarkers of Spinal Cord Injury Patients
Patients with spinal cord injury have a high prevalence of osteoporosis due to chronic skeletal unloading. Recently, various treatment drugs for osteoporosis have been developed. In particular, romosozumab, a sclerostin inhibitor, has been reported to have a high therapeutic effect as an inhibitor of bone resorption while promoting bone formation. …
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- 16 Feb, 2024
- 1 location