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Found 57 studies with search of: Open Studies | "Rare Diseases"
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Rank Status Study
1 Recruiting High Dose Intravenous N-Acetylcysteine Versus Iloprost for Early, Rapidly Progressive Diffuse Systemic Sclerosis
Condition: Scleroderma, Diffuse
Intervention: Drug: N-acetylcysteine (NAC)
2 Recruiting Canadian Fabry Disease Initiative (CFDI) Enzyme Replacement Therapy (ERT) Study
Condition: Fabry Disease
Interventions: Drug: agalsidase alfa (Replagal®) and agalsidase beta (Fabrazyme®);   Drug: Enzyme Replacement Therapy (ERT)
3 Recruiting Diagnosis and Treatment of Patients With Inborn Errors of Metabolism
Condition: Metabolism
Intervention:
4 Recruiting Absorption of Corticosteroids in Children With Juvenile Dermatomyositis
Conditions: Vasculitis, Hypersensitivity;   Connective Tissue Diseases;   Dermatomyositis;   Vasculitis
Interventions: Drug: Methylprednisolone;   Drug: Prednisolone
5 Recruiting The Experience of Uncertainty in Parents of Children With an Undiagnosed Medical Condition
Condition: Multiple Abnormalities
Intervention:
6 Recruiting Genetic Studies in the Amish and Mennonites
Condition: Genetic Disease
Intervention:
7 Recruiting Phase II Randomized Study of Physiologic Testosterone Replacement in Premenopausal, HIV-Positive Women
Conditions: HIV Infections;   Cachexia
Intervention: Drug: testosterone
8 Recruiting Measurement of Outcome of Surgical Treatment in Patients With Acromegaly
Condition: Acromegaly
Intervention:
9 Recruiting Study of Human Botulism Immunoglobulin in Infants With Botulism
Conditions: Infant Botulism;   Botulism
Intervention: Drug: botulism immune globulin
10 Recruiting Immunologic Evaluation in Patients With DiGeorge Syndrome or Velocardiofacial Syndrome
Conditions: DiGeorge Syndrome;   Shprintzen Syndrome;   Chromosome Abnormalities;   Abnormalities, Multiple;   Conotruncal Cardiac Defects
Intervention:
11 Recruiting Study of Clotrimazole and Hydroxyurea in Patients With Sickle Cell Syndromes
Condition: Sickle Cell Anemia
Interventions: Drug: clotrimazole;   Drug: hydroxyurea
12 Recruiting Genetic Study of Insulin-Like Growth Factor-I Receptor Mutations in Patients With Intrauterine Growth Retardation
Condition: Intrauterine Growth Retardation
Intervention:
13 Recruiting Clinical and Molecular Correlations in Spinocerebellar Ataxia Type 10 (SCA10)
Condition: Hereditary Ataxia
Intervention:
14 Recruiting Randomized Study of Human Parathyroid Hormone in Middle-Aged Men With Idiopathic Osteoporosis
Condition: Osteoporosis
Intervention: Drug: human parathyroid hormone
15 Recruiting Study of Treatment and Metabolism in Patients With Urea Cycle Disorders
Condition: Amino Acid Metabolism, Inborn Errors
Interventions: Behavioral: Protein and calorie controlled diet;   Gene Transfer: Ornithine transcarbamylase vector
16 Recruiting Phase II Randomized Study of Collagenase in Patients With Residual Type Dupuytren's Disease
Condition: Dupuytren's Contracture
Intervention: Drug: collagenase
17 Recruiting Randomized Study of Tauroursodeoxycholic Acid in Prophylactic Therapy of Total Parenteral Nutrition Associated Cholestasis in Infants
Condition: Cholestasis
Intervention: Drug: tauroursodeoxycholic acid
18 Recruiting Phase II Randomized Trial of Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers
Conditions: Skin Ulcers;   Sickle Cell Anemia
Intervention: Drug: arginine butyrate
19 Recruiting Phase II Randomized Study of Cultured Skin Substitutes Versus Split Thickness Skin Grafts in Patients With Severe Burn Injuries
Condition: Burns
Intervention: Procedure: Skin graft
20 Recruiting Randomized Study of Ketorolac in Infants Undergoing Surgery
Conditions: Pain;   Intraoperative Complications;   Postoperative Complications
Intervention: Drug: ketorolac

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