558 studies found for:    "succinic semialdehyde dehydrogenase deficiency" OR "brain diseases, metabolic, inborn"
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Rank Status Study
21 Recruiting Compare Pharmacokinetic(PK) Profiles of XZK vs Lovastatin in Healthy Male Volunteers
Condition: Lipid Metabolism Disorder
Interventions: Drug: XueZhiKang;   Drug: Lovastatin
22 Not yet recruiting A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy
Intervention: Dietary Supplement: vitamin D3
23 Recruiting Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)
Condition: Gaucher Disease Type 1
Intervention: Drug: N-acetylcysteine
24 Enrolling by invitation Fabry's Disease and Pregnancy (PREFAB)
Conditions: Fabry's Disease;   Pregnancy
Intervention: Other: Questionnaire
25 Not yet recruiting This Study is to Evaluate the Effect of Velaglucerase Alfa (VPRIV®) on Patients With Type 1 Gaucher Disease Through the IV Administration of VPRIV® Over 2 Years
Condition: Type 1 Gaucher Disease
Intervention: Drug: Velaglucerase alfa
26 Recruiting Metabolic Complications Following Knee Injury in Young and Middle Aged Patients
Conditions: Glucose Metabolism Disorders;   Lipid Metabolism Disorders;   Sedentary Lifestyle
Interventions: Biological: Blood and Urine sampling;   Radiation: DXA scan;   Biological: OGTT
27 Recruiting Evaluating the Potential of the Diet as Disease Modifier in Amyotrophic Lateral Sclerosis (JERN_ALS)
Conditions: Amyotrophic Lateral Sclerosis;   Dietary Habits;   Disorder of Fatty Acid Metabolism
Intervention:
28 Recruiting Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy
Conditions: Metachromatic Leukodystrophy;   Adrenoleukodystrophy
Intervention: Genetic: transduced CD34+ hematopoietic stem cell
29 Not yet recruiting Simplified Diet Approach in Phenylketonuria
Condition: Phenylketonuria
Intervention: Behavioral: Dietary Protein Counting and Free Fruits and Vegetables
30 Active, not recruiting Flank Study With Crown Cooling Insert
Condition: Body Fat Disorder
Intervention: Device: The ZELTIQ System
31 Not yet recruiting WILSTIM - DBS (WILson STIMulation - Deep Brain Stimulation)
Conditions: Severe Dystonia;   Wilson's Disease
Interventions: Device: Medtronic, Activa® PC "on";   Device: Medtronic, Activa® PC "off"
32 Recruiting A Dose-escalating Clinical Trial With KH176
Conditions: MELAS;   LHON;   Leigh Syndrome;   Mitochondrial Disease;   Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
Interventions: Drug: KH176;   Drug: placebo
33 Recruiting A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
Condition: Gaucher Disease
Intervention: Drug: eliglustat
34 Recruiting A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
Condition: Gaucher Disease
Intervention: Drug: eliglustat
35 Recruiting Skeletal Response to Eliglustat in Patients With Gaucher Disease
Condition: Gaucher's Disease
Intervention: Drug: eliglustat
36 Recruiting Study of 2-hydroxypropyl-β-cyclodextrin (VTS-270) to Treat Niemann-Pick Type C1 (NPC1) Disease
Condition: Niemann-Pick Disease, Type C
Interventions: Drug: VTS-270;   Drug: Sham Procedure Control
37 Completed Conjugated Linoleic Acid in Obese Women
Conditions: Lipid Metabolism Disorders;   Obesity
Interventions: Dietary Supplement: CLA;   Behavioral: Placebo
38 Recruiting The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
Conditions: Gaucher Disease Type 1;   Gaucher Disease Type 3
Intervention: Drug: Velaglucerase alfa
39 Not yet recruiting Prevalence of Pompe's Disease in Respiratory Clinics
Condition: Pompe's Disease
Intervention: Other: Prospective observational study
40 Recruiting Immune Modulation Therapy for Pompe Disease
Condition: Pompe Disease
Interventions: Drug: Rituximab;   Drug: intravenous immune globulin;   Drug: Bortezomib;   Drug: Methotrexate

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Indicates status has not been verified in more than two years