68 studies found for:    Open Studies | "Rare Diseases"
Show Display Options
Rank Status Study
21 Unknown  Immunologic Evaluation in Patients With DiGeorge Syndrome or Velocardiofacial Syndrome
Conditions: DiGeorge Syndrome;   Shprintzen Syndrome;   Chromosome Abnormalities;   Abnormalities, Multiple;   Conotruncal Cardiac Defects
Intervention:
22 Unknown  Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
Condition: Gaucher's Disease
Intervention: Drug: glucocerebrosidase
23 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:
24 Unknown  Phase I/II Randomized Study of Hydroxyurea With or Without Clotrimazole in Patients With Sickle Cell Anemia
Condition: Sickle Cell Anemia
Interventions: Drug: clotrimazole;   Drug: hydroxyurea
25 Unknown  Phase III Randomized Study of Oral Acyclovir in Infants With Herpes Simplex Virus Infection Involving the Central Nervous System
Condition: Herpes Simplex
Intervention: Drug: acyclovir
26 Unknown  Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease
Conditions: Gaucher's Disease;   Osteopenia
Interventions: Drug: alendronate sodium;   Drug: calcium carbonate;   Drug: cholecalciferol
27 Unknown  International Registry for Severe Chronic Neutropenia
Condition: Neutropenia
Intervention:
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  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
30 Unknown  Phase I Study of Vasoactive Intestinal Peptide in Patients With Acute Respiratory Distress Syndrome and Sepsis
Conditions: Sepsis;   Respiratory Distress Syndrome;   Respiratory Distress Syndrome, Adult
Intervention: Drug: vasoactive intestinal peptide
31 Unknown  Ultraviolet Light Therapy for Systemic Lupus Erythematosus
Condition: Systemic Lupus Erythematosus
Intervention: Procedure: ultraviolet A-1 light treatment
32 Unknown  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
33 Unknown  Phase II Study of Dietary Cholesterol for Smith-Lemli-Opitz Syndrome
Condition: Smith-Lemli-Opitz Syndrome
Intervention: Behavioral: diet
34 Unknown  Randomized Study of L-Baclofen in Patients With Refractory Trigeminal Neuralgia
Condition: Trigeminal Neuralgia
Intervention: Drug: L-baclofen
35 Unknown  Studies in Porphyria I: Characterization of Enzyme Defects
Condition: Porphyria
Intervention:
36 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:
37 Unknown  Study of Depression, Peptides, and Steroids in Cushing's Syndrome
Condition: Cushing's Syndrome
Intervention:
38 Unknown  Study of Recombinant Human Insulin-Like Growth Factor I in Patients With Severe Insulin Resistance
Conditions: Insulin Resistance;   Hyperglycemia
Intervention: Drug: insulin-like growth factor I
39 Unknown  Phase II Randomized Study of Collagenase in Patients With Residual Type Dupuytren's Disease
Condition: Dupuytren's Contracture
Intervention: Drug: collagenase
40 Unknown  Study of Systemic Amyloidosis Presentation and Prognosis
Condition: Amyloidosis
Intervention:

First Page    Show previous page of results Previous Page (1-20) Studies Shown (21-40) Next Page (41-60) Show next page of results    Last Page
Indicates status has not been verified in more than two years