66 studies found for:    Open Studies | "Rare Diseases"
Show Display Options
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 Riluzole in the Treatment of Spasticity in the Traumatic Chronic Spinal Cord Injury Condition
Condition: Spinal Cord Injury
Interventions: Drug: Riluzole;   Drug: Placebo;   Biological: Blood Samples
25 Recruiting Slowing Down Disease Progression in Premanifest SCA: a Piloting Interventional Exergame Trial
Condition: Spinocerebellar Ataxia
Intervention: Other: Motor training
26 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®)
27 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
28 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
29 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
30 Recruiting Efficacy and Safety Study of I10E in Treatment of Patients With CIDP
Condition: Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Intervention: Drug: I10E
31 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:
32 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)
33 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
34 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:
35 Unknown  Study of Energy Requirements in Critically Ill Newborns
Condition: Respiratory Distress Syndrome
Intervention:
36 Unknown  Study of Energy Expenditure in Infants With Ventricular Septal Defects
Condition: Heart Septal Defects, Ventricular
Intervention:
37 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
38 Unknown  Randomized Study of Testosterone and Progressive Resistance Exercise in Men With Burn Injury
Condition: Burns
Intervention: Drug: testosterone
39 Unknown  Study of Neurobehavioral Outcome in Children or Adolescents With Closed Head Injuries
Condition: Head Injuries, Closed
Intervention:
40 Unknown  Diagnostic Study of Quantitative Imaging and Spectroscopy in Patients With Multiple Sclerosis
Condition: Multiple Sclerosis
Intervention: Drug: standard gadolinium contrast

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