549 studies found for:    "succinic semialdehyde dehydrogenase deficiency" OR "brain diseases, metabolic, inborn"
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Rank Status Study
21 Not yet recruiting Simplified Diet Approach in Phenylketonuria
Condition: Phenylketonuria
Intervention: Behavioral: Dietary Protein Counting and Free Fruits and Vegetables
22 Recruiting Flank Study With Crown Cooling Insert
Condition: Body Fat Disorder
Intervention: Device: The ZELTIQ System
23 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"
24 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
25 Recruiting A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
Condition: Gaucher Disease
Intervention: Drug: eliglustat
26 Recruiting A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
Condition: Gaucher Disease
Intervention: Drug: eliglustat
27 Recruiting Skeletal Response to Eliglustat in Patients With Gaucher Disease
Condition: Gaucher's Disease
Intervention: Drug: eliglustat
28 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
29 Completed Conjugated Linoleic Acid in Obese Women
Conditions: Lipid Metabolism Disorders;   Obesity
Interventions: Dietary Supplement: CLA;   Behavioral: Placebo
30 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
31 Not yet recruiting Prevalence of Pompe's Disease in Respiratory Clinics
Condition: Pompe's Disease
Intervention: Other: Prospective observational study
32 Recruiting Immune Modulation Therapy for Pompe Disease
Condition: Pompe Disease
Interventions: Drug: Rituximab;   Drug: intravenous immune globulin;   Drug: Bortezomib;   Drug: Methotrexate
33 Recruiting Miglustat on Gaucher Disease Type IIIB
Condition: Gaucher Disease
Interventions: Drug: Miglustat;   Drug: ERT
34 Recruiting Intervention and Outcomes in Duarte Galactosemia
Condition: Duarte Galactosemia
35 Enrolling by invitation Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease
Condition: Fabry Disease
Intervention: Drug: GZ/SAR402671
36 Recruiting Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)
Condition: Urea Cycle Disorders
Intervention: Biological: HepaStem
37 Recruiting Impact of Enzyme Replacement Therapy on Cardiac Function in Patients With Fabry's Cardiomyopathy (RECAFTURE Trial)
Condition: Fabry's Disease
38 Enrolling by invitation A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
Condition: Phenylketonuria
Intervention: Other: Administration of CANTAB and Subject Global Assessment
39 Recruiting Prevalence of Fabry's Disease in a Population of Patients With Chronic Pain
Conditions: Fabry's Disease;   Chronic Pain
Intervention: Genetic: Diagnosis of Fabry disease
40 Enrolling by invitation Testing of Four Home Phenylalanine Monitoring Prototype Devices
Condition: Phenylketonurias

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Indicates status has not been verified in more than two years