69 studies found for:    Open Studies | "Rare Diseases"
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Rank Status Study
21 Unknown  Randomized Study of Photodynamic Therapy Using Dihematoporphyrin in Patients With Corneal Neovascularization
Condition: Corneal Neovascularization
Interventions: Drug: Dihematoporphyrin derivative;   Drug: prednisolone;   Procedure: Laser surgery
22 Recruiting Hereditary Inclusion Body Myopathy-Disease Monitoring (HIBM-DMP): A Combined Registry and Prospective Observational Natural History Study to Assess HIBM Disease
Conditions: Hereditary Inclusion Body Myopathy;   GNE Myopathy;   Nonaka Disease;   Quadriceps Sparing Myopathy (QSM);   Distal Myopathy With Rimmed Vacuoles (DMRV)
Intervention:
23 Recruiting Study of Total Body Irradiation in Combination With Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation Followed By Cyclosporine and Mycophenolate Mofetil in High Risk-Patients With Human Immunodeficiency Virus-1
Condition: HIV Infections
Interventions: Drug: Cyclosporine;   Drug: Mycophenolate mofetil;   Procedure: Total body irradiation and stem cell or bone marrow transplantation
24 Unknown  Randomized Study of Folic Acid Therapy for Hyperhomocysteinemia in Patients With End Stage Renal Disease Receiving Hemodialysis
Conditions: End Stage Renal Disease;   Hyperhomocysteinemia
Interventions: Drug: cyanocobalamin;   Drug: folic acid;   Drug: pyridoxine
25 Unknown  Diagnostic Study of Quantitative Imaging and Spectroscopy in Patients With Multiple Sclerosis
Condition: Multiple Sclerosis
Intervention: Drug: standard gadolinium contrast
26 Unknown  International Registry for Severe Chronic Neutropenia
Condition: Neutropenia
Intervention:
27 Unknown  Study of Bile Acids in Patients With Peroxisomal Disorders
Conditions: Infantile Refsum's Disease;   Zellweger Syndrome;   Bifunctional Enzyme Deficiency;   Adrenoleukodystrophy
Interventions: Drug: chenodeoxycholic acid;   Drug: cholic acid;   Drug: ursodiol
28 Unknown  Phase III Randomized Study of Collagenase in Patients With Residual Stage Dupuytren's Disease
Condition: Dupuytren's Contracture
Intervention: Drug: collagenase
29 Unknown  Leuprolide in Determining the Cause of Gonadotropin Deficiency
Condition: Hypogonadism
Interventions: Drug: gonadotropin releasing hormone;   Drug: leuprolide
30 Unknown  Study of Clotrimazole and Hydroxyurea in Patients With Sickle Cell Syndromes
Condition: Sickle Cell Anemia
Interventions: Drug: clotrimazole;   Drug: hydroxyurea
31 Unknown  Study of Energy Expenditure in Infants With Ventricular Septal Defects
Condition: Heart Septal Defects, Ventricular
Intervention:
32 Unknown  Immunologic Evaluation in Patients With DiGeorge Syndrome or Velocardiofacial Syndrome
Conditions: DiGeorge Syndrome;   Shprintzen Syndrome;   Chromosome Abnormalities;   Abnormalities, Multiple;   Conotruncal Cardiac Defects
Intervention:
33 Unknown  Phase II Study of Dietary Cholesterol for Smith-Lemli-Opitz Syndrome
Condition: Smith-Lemli-Opitz Syndrome
Intervention: Behavioral: diet
34 Unknown  Study of Tauroursodeoxycholic Acid for Hepatobiliary Disease in Cystic Fibrosis
Condition: Cystic Fibrosis
Interventions: Drug: tauroursodeoxycholic acid;   Drug: ursodiol
35 Unknown  Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
Condition: Gaucher's Disease
Intervention: Drug: glucocerebrosidase
36 Unknown  Genetic Study of the FBN1 Gene and Fibrillin-1 Abnormalities in Choctaw Native Americans and Other Patients With Systemic Sclerosis
Condition: Systemic Sclerosis
Intervention:
37 Unknown  Randomized Study of L-Baclofen in Patients With Refractory Trigeminal Neuralgia
Condition: Trigeminal Neuralgia
Intervention: Drug: L-baclofen
38 Unknown  Studies in Porphyria I: Characterization of Enzyme Defects
Condition: Porphyria
Intervention:
39 Unknown  Study of Human Botulism Immunoglobulin in Infants With Botulism
Conditions: Infant Botulism;   Botulism
Intervention: Drug: botulism immune globulin
40 Unknown  Study of Neurobehavioral Outcome in Children or Adolescents With Closed Head Injuries
Condition: Head Injuries, Closed
Intervention:

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Indicates status has not been verified in more than two years