64 studies found for:    Open Studies | "Rare Diseases"
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Rank Status Study
21 Recruiting Canadian Fabry Disease Initiative (CFDI) Enzyme Replacement Therapy (ERT) Study
Condition: Fabry Disease
Interventions: Drug: agalsidase alfa;   Drug: Agalsidase beta
22 Recruiting Treatment Alternatives in iSGS (NoAAC PR-02 Study)
Condition: Idiopathic Subglottic Stenosis (iSGS)
Interventions: Procedure: Endoscopic dilation of subglottic stenosis;   Procedure: Endoscopic resection of the stenosis;   Procedure: Tracheal Resection
23 Recruiting Reduction in Splanchnic Capacitance Contributes to Sympathetically Dependent Hypertension in Autonomic
Conditions: Multiple System Atrophy;   Orthostatic Hypotension;   Supine Hypertension
Interventions: Drug: Trimethaphan;   Drug: Placebo
24 Not yet recruiting Therapeutic Anticoagulation Strategy for Acute Chest Syndrome
Conditions: Anemia;   Sickle Cell;   Acute Chest Syndrome;   Low-Molecular-Weight Heparin
Interventions: Drug: Prophylactic anticoagulation ( INNOHEP®);   Drug: Curative anticoagulation ( INNOHEP®)
25 Recruiting A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7
Conditions: Sly Syndrome;   MPS VII;   Mucopolysaccharidosis;   Mucopolysaccharidosis VII
Intervention: Drug: UX003
26 Recruiting An Open-Label Study of UX003-rhGUS Enzyme Replacement Treatment in MPS 7 Patients Less Than 5 Years of Age
Conditions: Sly Syndrome;   MPS VII;   Mucopolysaccharidosis;   Mucopolysaccharidosis VII
Intervention: Drug: UX003
27 Recruiting Efficacy and Safety Study of I10E in the Maintenance Treatment of Patients With CIDP: Extension of PRISM Study I10E-1302
Condition: Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Intervention: Drug: I10E
28 Recruiting Efficacy and Safety Study of I10E in Treatment of Patients With CIDP
Condition: Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Intervention: Drug: I10E
29 Recruiting GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
Conditions: Hereditary Inclusion Body Myopathy;   GNE Myopathy;   Nonaka Disease;   Quadriceps Sparing Myopathy (QSM);   Distal Myopathy With Rimmed Vacuoles (DMRV)
Intervention:
30 Recruiting Evaluation of an Advanced Lower Extremity Neuroprostheses
Conditions: Spinal Cord Injuries;   Stroke;   Paralysis;   Tetraplegia;   Paraplegia
Intervention: Device: IST-16 (16-channel implanted stimulator-telemeter)
31 Unknown  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
32 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:
33 Unknown  Study of Energy Requirements in Critically Ill Newborns
Condition: Respiratory Distress Syndrome
Intervention:
34 Unknown  Study of Energy Expenditure in Infants With Ventricular Septal Defects
Condition: Heart Septal Defects, Ventricular
Intervention:
35 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
36 Unknown  Randomized Study of Testosterone and Progressive Resistance Exercise in Men With Burn Injury
Condition: Burns
Intervention: Drug: testosterone
37 Unknown  Study of Neurobehavioral Outcome in Children or Adolescents With Closed Head Injuries
Condition: Head Injuries, Closed
Intervention:
38 Unknown  Diagnostic Study of Quantitative Imaging and Spectroscopy in Patients With Multiple Sclerosis
Condition: Multiple Sclerosis
Intervention: Drug: standard gadolinium contrast
39 Unknown  Gluconeogenesis in Very Low Birth Weight Infants Who Are Receiving Nutrition By Intravenous Infusion
Conditions: Infant, Low Birth Weight;   Hyperglycemia
Interventions: Drug: alanine;   Drug: amino acids;   Drug: glucagon;   Drug: glucose;   Drug: glutamine;   Drug: glycerol;   Drug: insulin;   Drug: leucine;   Drug: lipids;   Drug: urea
40 Unknown  Genetic Study of Insulin-Like Growth Factor-I Receptor Mutations in Patients With Intrauterine Growth Retardation
Condition: Intrauterine Growth Retardation
Intervention:

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Indicates status has not been verified in more than two years