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Found 59 studies with search of: Open Studies | "Rare Diseases"
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Rank Status Study
1 Recruiting Nephrotic Syndrome Study Network
Conditions: Minimal Change Disease;   Membranous Nephropathy;   Glomerulosclerosis, Focal Segmental
Intervention:
2 Unknown † High Dose Intravenous N-Acetylcysteine Versus Iloprost for Early, Rapidly Progressive Diffuse Systemic Sclerosis
Condition: Scleroderma, Diffuse
Intervention: Drug: N-acetylcysteine (NAC)
3 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)
4 Recruiting Pulmonary Hypertension, Quality of Life and Psychosocial Factors
Condition: Pulmonary Hypertension
Intervention: Behavioral: Evaluation of quality of life
5 Recruiting Follow-up Protocol for Patients With Cancer/AIDS/Skin Disease
Conditions: Acquired Immunodeficiency Syndrome;   Neoplasm;   Skin Disease;   HIV Infections
Intervention:
6 Recruiting Cure Cystinosis International Registry
Conditions: Cystinosis;   Nephropathic Cystinosis;   Renal Fanconi Syndrome
Intervention:
7 Recruiting Diagnosis and Treatment of Patients With Inborn Errors of Metabolism
Condition: Metabolism
Intervention:
8 Recruiting Prospective Collection of Data in Patients With Peripheral T-Cell Lymphoma
Condition: Lymphoma, T-Cell, Peripheral
Intervention:
9 Recruiting Genetic Studies in the Amish and Mennonites
Condition: Genetic Disease
Intervention:
10 Recruiting Cross-sectional Characterization of Idiopathic Bronchiectasis
Conditions: Idiopathic Bronchiectasis;   Nontuberculous Mycobacterium (NTM) Infection
Intervention:
11 Unknown † Phase II Randomized Study of Physiologic Testosterone Replacement in Premenopausal, HIV-Positive Women
Conditions: HIV Infections;   Cachexia
Intervention: Drug: testosterone
12 Unknown † Measurement of Outcome of Surgical Treatment in Patients With Acromegaly
Condition: Acromegaly
Intervention:
13 Unknown † Study of Human Botulism Immunoglobulin in Infants With Botulism
Conditions: Infant Botulism;   Botulism
Intervention: Drug: botulism immune globulin
14 Unknown † Immunologic Evaluation in Patients With DiGeorge Syndrome or Velocardiofacial Syndrome
Conditions: DiGeorge Syndrome;   Shprintzen Syndrome;   Chromosome Abnormalities;   Abnormalities, Multiple;   Conotruncal Cardiac Defects
Intervention:
15 Unknown † Study of Clotrimazole and Hydroxyurea in Patients With Sickle Cell Syndromes
Condition: Sickle Cell Anemia
Interventions: Drug: clotrimazole;   Drug: hydroxyurea
16 Unknown † Genetic Study of Insulin-Like Growth Factor-I Receptor Mutations in Patients With Intrauterine Growth Retardation
Condition: Intrauterine Growth Retardation
Intervention:
17 Unknown † Clinical and Molecular Correlations in Spinocerebellar Ataxia Type 10 (SCA10)
Condition: Hereditary Ataxia
Intervention:
18 Unknown † Randomized Study of Human Parathyroid Hormone in Middle-Aged Men With Idiopathic Osteoporosis
Condition: Osteoporosis
Intervention: Drug: human parathyroid hormone
19 Unknown † Study of Treatment and Metabolism in Patients With Urea Cycle Disorders
Condition: Amino Acid Metabolism, Inborn Errors
Interventions: Behavioral: Protein and calorie controlled diet;   Genetic: Ornithine transcarbamylase vector
20 Unknown † Phase II Randomized Study of Collagenase in Patients With Residual Type Dupuytren's Disease
Condition: Dupuytren's Contracture
Intervention: Drug: collagenase

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† Indicates status has not been verified in more than two years
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