58 studies found for:    Open Studies | "Rare Diseases"
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Rank Status Study
21 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
22 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
23 Not yet 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
24 Recruiting Efficacy and Safety Study of I10E in Treatment of Patients With CIDP
Condition: Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Intervention: Drug: I10E
25 Recruiting Efficacy and Safety of Ofatumumab in Treatment of Pemphigus Vulgaris
Condition: Pemphigus
Interventions: Biological: Ofatumumab;   Biological: Placebo
26 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)
27 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)
28 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
29 Unknown  Genetic Study of the FBN1 Gene and Fibrillin-1 Abnormalities in Choctaw Native Americans and Other Patients With Systemic Sclerosis
Condition: Systemic Sclerosis
30 Unknown  Study of Energy Requirements in Critically Ill Newborns
Condition: Respiratory Distress Syndrome
31 Unknown  Study of Energy Expenditure in Infants With Ventricular Septal Defects
Condition: Heart Septal Defects, Ventricular
32 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
33 Unknown  Randomized Study of Testosterone and Progressive Resistance Exercise in Men With Burn Injury
Condition: Burns
Intervention: Drug: testosterone
34 Unknown  Study of Neurobehavioral Outcome in Children or Adolescents With Closed Head Injuries
Condition: Head Injuries, Closed
35 Unknown  Diagnostic Study of Quantitative Imaging and Spectroscopy in Patients With Multiple Sclerosis
Condition: Multiple Sclerosis
Intervention: Drug: standard gadolinium contrast
36 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
37 Unknown  Genetic Study of Insulin-Like Growth Factor-I Receptor Mutations in Patients With Intrauterine Growth Retardation
Condition: Intrauterine Growth Retardation
38 Unknown  Immunologic Evaluation in Patients With DiGeorge Syndrome or Velocardiofacial Syndrome
Conditions: DiGeorge Syndrome;   Shprintzen Syndrome;   Chromosome Abnormalities;   Abnormalities, Multiple;   Conotruncal Cardiac Defects
39 Unknown  Measurement of Outcome of Surgical Treatment in Patients With Acromegaly
Condition: Acromegaly
40 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

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Indicates status has not been verified in more than two years