NCT03362164
- Conditions
- Aortic Stenosis, Subvalvular, Aortic Valve Disease, Aortic Valve Stenosis, +43 more
- Locations
-
- Wuerzburg University Hospital Würzburg, Bavaria, Germany
Clinical trial condition
Lysosomal Storage Diseases, Nervous System
Explore clinical trials studying Lysosomal Storage Diseases, Nervous System. Study availability and eligibility vary by location and protocol.
520 public trials
102 recruiting studies
Lysosomal Storage Diseases, Nervous System trial results
NCT02399748
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
- Locations
-
- National Taiwan University Hospital Taipei, Taiwan
Not yet recruiting
Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD
NCT07046338
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- Shenzhen Geno-Immune Medical Institute Shenzhen, Guangdong, China
Not yet recruiting
4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma
NCT04637503
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +24 more
- Locations
-
- Shenzhen Geno-Immune Medical Institute Shenzhen, Guangzhou, China
Not yet recruiting
Direct Lentiviral Injection Gene Therapy for MLD
NCT03725670
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- Lung-Ji Chang Shenzhen, Guangdong, China
Not yet recruiting
Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial
NCT07479953
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
Not specified
Not yet recruiting
A Study of S-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD)
NCT07787650
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
- Locations
-
Not specified
Not yet recruiting
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
NCT07778667
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
Not specified
Not yet recruiting
An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)
NCT07750990
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
- Locations
-
Not specified
Not yet recruiting
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)
NCT07740512
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +24 more
- Locations
-
Not specified