1809 studies found for:    Open Studies | "Genetic Diseases, Inborn"
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21 Not yet recruiting AUgmented REality for the Visually Impaired - Part 1
Conditions: Retinitis Pigmentosa;   Glaucoma
Interventions: Procedure: Find visual comfort threshold related to light intensity;   Procedure: Find the size of the visual field;   Procedure: Effectiveness of brightness control;   Procedure: Performance of color correction;   Device: Vuzix Wrap 1200DX virtural reality glasses
22 Not yet recruiting Safety, Efficacy, and Feasibility of High-dose Cholecalciferol in Pediatric Patients With Cystic Fibrosis
Condition: Vitamin D Deficiency
Intervention: Drug: Treatment
23 Not yet recruiting Rocky Mountain Alzheimer's Disease Center Longitudinal Biomarker and Clinical Phenotyping Study
Conditions: Alzheimer Disease;   Down Syndrome;   Mild Cognitive Impairment
Intervention: Other: No Intervention(s)
24 Not yet recruiting Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C
Condition: Niemann-Pick Disease, Type C
Interventions: Drug: arimoclomol;   Drug: Placebo
25 Not yet recruiting Post-Marketing Non-Interventional Safety Evaluation of Obizur in the Treatment of Bleeding Episodes for Patients With Acquired Hemophilia A
Condition: Acquired Hemophilia A
Intervention: Biological: OBIZUR
26 Not yet recruiting Effect of IFN-γ on Innate Immune Cells
Condition: Chronic Granulomatous Disease
Intervention: Drug: Administration of drug (Interferon-gamma 1-b) subcutaneously
27 Recruiting Nerve Growth Factor Eye Drops Treatment in Patients With Retinitis Pigmentosa and Cystoid Macular Edema
Conditions: Retinitis Pigmentosa;   Cystoid Macular Edema
Interventions: Drug: rhNGF 180 µg/ml eye drops solution;   Drug: vehicle eye drops
28 Recruiting The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia
Conditions: Nausea;   Vomiting
Interventions: Drug: Dronabinol;   Other: Placebo
29 Recruiting Survey in a Population of Sickle Cell Disease Patients to Evaluate the Transition Between the Queen Fabiola Children Hospital and the CHU Brugmann Hospital, and the Quality of the Hospital Care Within the CHU Brugmann Hospital.
Condition: Sickle Cell Disease
Intervention: Other: Survey
30 Recruiting Hyperpolarized Xenon MRI in Cystic Fibrosis Pulmonary Exacerbations
Condition: Cystic Fibrosis
31 Recruiting Trial of FG-3019, a Monoclonal Antibody to CTGF, in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: FG-3019
32 Not yet recruiting Minimize Menorrhagia in Women With Type 1 Von Willebrand Disease
Condition: Type 1 Von Willebrand Disease
Interventions: Drug: recombinant von Willebrand factor;   Drug: tranexamic acid
33 Not yet recruiting Trial to Demonstrate the Efficacy and Safety of Internet-delivered Behavioral Treatment for Adults With Tic Disorders
Conditions: Tics;   Tourette Syndrome
Interventions: Behavioral: (i)Comprehensive Behavioral Intervention for Tics;   Behavioral: internet-delivered psychoeducation and relaxation training
34 Recruiting SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease
Condition: Gaucher Disease
35 Not yet recruiting Safety, Tolerability and Pharmacokinetics Study of AIR DNAse Administered by Inhalation to Healthy Adult Volunteers
Condition: Cystic Fibrosis (CF)
Interventions: Drug: AIR DNase;   Drug: Placebo
36 Recruiting Hepatitis B in Cystic Fibrosis and Latent Tuberculosis Respectively
Conditions: Cystic Fibrosis;   Hepatitis A;   Hepatitis B;   Tuberculosis
Intervention: Biological: Vaccination with vaccine against hepatitis A and B
37 Not yet recruiting An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia
Conditions: Erythrocyte Transfusion;   Beta-Thalassemia
Interventions: Drug: Luspatercept;   Other: Placebo
38 Not yet recruiting Multi-center Study of SC411 for Sickle Cell Disease
Condition: Sickle Cell Disease
Interventions: Drug: SC411;   Drug: Placebo
39 Not yet recruiting Effects of Botulinum Toxin Injections in Patients With Hereditary Spastic Paraplegia
Condition: Hereditary Spastic Paraplegia
Interventions: Other: Botulinum Toxin Injections;   Other: Placebo Injections
40 Recruiting Compare Pharmacokinetic(PK) Profiles of XZK vs Lovastatin in Healthy Male Volunteers
Condition: Lipid Metabolism Disorder
Interventions: Drug: XueZhiKang;   Drug: Lovastatin

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Indicates status has not been verified in more than two years