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41 studies found for:    Open Studies | "Rare Diseases"
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Rank Status Study
21 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
22 Recruiting Natural History and Advanced Genetic Study of Pyruvate Dehydrogenase Complex Deficiencies
Condition: Pyruvate Dehydrogenase Complex Deficiency Disease
Intervention: Other: No intervention
23 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
24 Recruiting Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda
Condition: Porphyria Cutanea Tarda
Interventions: Drug: Hydroxychloroquine;   Procedure: Phlebotomy
25 Recruiting Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network
Condition: Undiagnosed Disease
Intervention:
26 Recruiting Pyrimethamine, Sulfadiazine, and Leucovorin in Treating Patients With Congenital Toxoplasmosis
Condition: Toxoplasmosis
Interventions: Drug: Leucovorin calcium;   Drug: Pyrimethamine;   Drug: Spiramycin;   Drug: Sulfadiazine
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 Williams Syndrome (WS) and Supravalvular Aortic Stenosis (SVAS) DNA and Tissue Bank
Conditions: Williams Syndrome;   Supravalvular Aortic Stenosis;   Cardiovascular Disease
Intervention:
30 Recruiting Human Biospecimen Procurement Protocol: Biorepository to Support Translational Research to Identify Disease Mechanism(s)
Conditions: Undiagnosed Diseases;   Cardiovascular Diseases
Intervention:
31 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®)
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 Recruiting Slowing Down Disease Progression in Premanifest SCA: a Piloting Interventional Exergame Trial
Condition: Spinocerebellar Ataxia
Intervention: Other: Motor training
34 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
35 Recruiting Diagnosis and Treatment of Patients With Inborn Errors of Metabolism
Condition: Metabolism
Intervention:
36 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:
37 Recruiting Cure Cystinosis International Registry
Conditions: Cystinosis;   Nephropathic Cystinosis;   Renal Fanconi Syndrome
Intervention:
38 Recruiting T-Cell Project: Prospective Collection of Data in Patients With Peripheral T-Cell Lymphoma
Condition: Lymphoma, T-Cell, Peripheral
Intervention:
39 Recruiting European Calciphylaxis Registry Network
Conditions: Calcific Uremic Arteriolopathy (CUA);   Calciphylaxis
Intervention:
40 Recruiting Phase 2 Study With SNF472 in Calciphylaxis Patients
Conditions: Calciphylaxis;   Calcific Uremic Arteriolopathy
Intervention: Drug: SNF472

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Study has passed its completion date and status has not been verified in more than two years.