165 studies found for:    Open Studies | "Brain Diseases, Metabolic, Inborn"
Show Display Options
Rank Status Study
21 Not yet recruiting Diet and Exercise in Pompe Disease
Conditions: Pompe Disease;   Glycogen Storage Disease Type II;   Lysosomal Storage Diseases
Intervention: Other: Diet and Exercise
22 Not yet recruiting A Pilot Study of Pyridostigmine in Pompe Disease
Condition: Pompe Disease
Intervention: Drug: Pyridostigmine Bromide
23 Recruiting Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
Condition: Pompe Disease
Interventions: Other: Thoracic MRI;   Other: Spirometry;   Other: Inspiratory Load Compensation;   Other: Maximal Inspiratory Pressure;   Other: Resting Breathing Pattern;   Other: Respiratory Muscle Endurance Test
24 Recruiting Response to Diaphragmatic Pacing in Subjects With Pompe Disease
Condition: Pompe Disease
Interventions: Other: Pulmonary function testing;   Other: Resting breathing pattern;   Other: Respiratory muscle endurance test;   Other: Phrenic nerve stimulation;   Other: Maximal inspiratory pressure;   Other: Forced expiratory testing;   Other: EMG
25 Recruiting Distal Thigh Study
Condition: Body Fat Disorder
Intervention: Device: The ZELTIQ System
26 Recruiting The Metabolic Effect of Walnuts in Healthy Subjects
Condition: Lipid Metabolism Disorders
Intervention: Other: walnuts
27 Recruiting Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1
Condition: Hereditary Tyrosinemia, Type I
Intervention: Drug: Nitisinone
28 Recruiting Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care
Condition: Hereditary Tyrosinemia, Type I
Intervention: Drug: Nitisinone
29 Recruiting Genes Involved in Lipid Disorders
Condition: Dyslipidemia
Intervention:
30 Recruiting Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment
Condition: Urea Cycle Disorders
Intervention:
31 Recruiting A High-Resolution Magnetic Resonance Imaging Study to Evaluate the Effect of Rosuvastatin on Carotid Atherosclerotic Plaques
Conditions: Arteriosclerosis;   Diabetes Mellitus;   Lipid Disorder
Interventions: Drug: Rosuvastatin 5mg;   Drug: Rosuvastatin 20mg
32 Recruiting Biomarker for Mucolipidosis Disorder Type I, II, III or IV
Conditions: Mucolipidosis Type I;   Mucolipidosis Type II;   Mucolipidosis Type III;   Mucolipidosis Type IV;   Mucolipidosis
Intervention:
33 Recruiting Biomarker for GM1/GM2 - Gangliosidoses
Conditions: Gangliosidosis;   GM1-Gangliosidosis;   GM2-Gangliosidosis;   Hexosaminidase Activator Deficiency;   Tay-Sachs Disease, AB Variant;   Hexosaminidase A and B Deficiency;   Sandhoff Disease
Intervention:
34 Recruiting Biomarker for Farber Disease
Conditions: Farber's Lipogranulomatosis;   Ceramidase Deficiency;   Farber Disease;   Acid Ceramidase Deficiency;   AC Deficiency
Intervention:
35 Recruiting PKU Kuvan Imaging Study
Condition: Phenylketonuria
Intervention:
36 Recruiting Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of rhASM in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
Condition: Sphingomyelin Lipidosis
Intervention: Drug: GZ402665
37 Recruiting Efficacy and Safety Study of WTX101 in Adult Wilson Disease Patients
Condition: Wilson Disease
Interventions: Drug: WTX101;   Drug: Omeprazole
38 Recruiting The Impact of Artichoke Leaf Extract on Blood Cholesterol: Primary Study
Condition: Lipid Metabolism Disorders
Interventions: Dietary Supplement: Artichoke Leaf Extract;   Other: Placebo
39 Recruiting Nitric Oxide Supplementation in Argininosuccinic Aciduria
Conditions: Argininosuccinic Aciduria;   Argininosuccinate Lyase Deficiency;   Urea Cycle Disorders
Interventions: Dietary Supplement: Nitric oxide supplement;   Dietary Supplement: Placebo
40 Recruiting ExAblate Transcranial MRgFUS for the Management of Treatment-Refractory Movement Disorders
Conditions: Movement Disorders;   Essential Tremor;   Holmes Tremor;   Parkinson's Disease;   Wilson's Disease;   Huntington's Disease;   Dystonia;   Tardive Dyskinesia;   Orofacial Dyskinesias
Intervention: Device: Transcranial ExAblate System

First Page    Show previous page of results Previous Page (1-20) Studies Shown (21-40) Next Page (41-60) Show next page of results    Last Page
Indicates status has not been verified in more than two years