1765 studies found for:    Open Studies | "Genetic Diseases, Inborn"
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Rank Status Study
21 Recruiting Studies of Children With Metabolic and Other Genetic Disorders
Conditions: Genetic Disorder;   Metabolic Disease
Intervention:
22 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:
23 Recruiting Atherosclerosis in Familial Hypercholesterolemia
Condition: Familial Hypercholesterolemia
Intervention: Other: CT coronary angiogram and carotid ultrasound
24 Not yet recruiting Functional and Structural Imaging and Motor Control in Spinocerebellar Ataxia
Condition: Spinocerebellar Ataxia
Interventions: Behavioral: Error-reduction;   Behavioral: International Cooperative Ataxia Rating Scale;   Behavioral: Scale for the Assessment and Rating of Ataxia
25 Not yet recruiting HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)
Conditions: Duchenne Muscular Dystrophy;   Cardiomyopathy
Intervention: Drug: Allogeneic Cardiosphere-Derived Cells (CAP-1002)
26 Not yet recruiting A Study of RG1662 in Down Syndrome Among Children 6 to 11 Years of Age
Condition: Down Syndrome
Interventions: Drug: Placebo;   Drug: RG1662
27 Not yet recruiting Study of Recombinant Factor VIIa Fusion Protein (rVIIa-FP, CSL689) for On-demand Treatment of Bleeding Episodes in Patients With Hemophilia A or B With Inhibitors
Conditions: Hemophilia A With Inhibitors;   Hemophilia B With Inhibitors
Interventions: Drug: CSL689;   Drug: Eptacog alfa (activated)
28 Not yet recruiting GOLD-B: A Gene Therapy Study for Hemophilia B
Condition: Hemophilia B
Intervention: Genetic: SPK-9001
29 Recruiting Single Dose Study in Subjects With Severe Hemophilia A Comparing Pharmacokinetic Parameters for BAY81-8973 and Advate
Condition: Hemophilia A
Interventions: Drug: BAY81-8973;   Drug: Advate
30 Not yet recruiting A Study to Evaluate the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell Disease
Condition: Sickle Cell Disease
Interventions: Drug: Ticagrelor;   Drug: Placebo;   Drug: Analgesic
31 Recruiting Feasibility and Dose Tolerability of HD-tDCS in Healthy Adults and Adults With Down Syndrome
Condition: Down Syndrome
Intervention: Device: High definition transcranial direct current stimulation
32 Not yet recruiting VX15/2503 Treatment for Huntington Disease
Condition: Huntington Disease
Interventions: Drug: VX15/2503;   Drug: Placebo
33 Not yet recruiting Is Iron Deficiency the Cause of Anemia Among Women in Cambodia?
Conditions: Anemia;   Iron Deficiency;   Hemoglobin Disorder;   Infection;   Inflammation
Interventions: Dietary Supplement: Multiple micronutrients;   Dietary Supplement: Iron;   Dietary Supplement: Placebo
34 Recruiting Outpatient Antibiotic Treatment for a Cystic Fibrosis Pulmonary Exacerbation
Condition: Cystic Fibrosis
Intervention:
35 Recruiting Reliability of Functional Outcome Measures in Neurofibromatosis 1
Condition: Neurofibromatosis 1, NF1
Intervention:
36 Recruiting Safety/Efficacy Study to Assess Whether FVIII/VWF Concentrate Can Induce Immune Tolerance in Haemophilia A Patients
Condition: Hemophilia A
Intervention: Drug: Plasma-derived FVIII/VWF concentrate
37 Not yet recruiting A Two-Part Multicenter Prospective Longitudinal Study of CFTR-dependent Disease Profiling in Cystic Fibrosis (PROSPECT)
Condition: Cystic Fibrosis
Intervention: Other: Observational
38 Recruiting A Prospective Study to Collect High-Quality Documentation of Bleeds Health-Related Quality of Life (HRQoL), and Safety Outcomes in Patients With Hemophilia A and Factor VIII (FVIII) Inhibitors Treated With Standard-of-Care Treatment
Condition: Hemophilia A
Intervention: Drug: Bypassing Agents
39 Recruiting A Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency
Condition: Pyruvate Kinase Deficiency
Intervention: Drug: AG-348
40 Not yet recruiting SMART Mobile Application Technology Utilization in the Treatment of Sickle Cell Disease Post Day Hospital Discharge
Condition: Sickle Cell Disease
Intervention: Device: SMART app

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Indicates status has not been verified in more than two years