54 studies found for:    Open Studies | "Rare Diseases"
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Rank Status Study
1 Recruiting Pregnancy and Medically Assisted Conception in Rare Diseases
Conditions: Rheumatoid Arthritis;   Spondyloarthritis;   Psoriatic Arthritis;   Systemic Lupus Erythematosus;   Antiphospholipid Syndrome;   Sjogren Syndrome;   Scleroderma;   Myositis;   Vasculitis;   Mastocytosis;   Various Autoimmune and/or Systemic and/or Rare Diseases
Intervention:
2 Recruiting Rare Disease Patient Registry: Coordination of Rare Diseases at Sanford
Conditions: Rare Disorders;   Undiagnosed Disorders;   Disorders of Unknown Prevalence
Intervention:
3 Recruiting Orodental Manifestations of Rare Diseases
Condition: Rare Disease Orodontal
Intervention: Other: Salivary and blood samples
4 Recruiting Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in GRD
Condition: Genetic Rare Disease
Intervention: Drug: Busulfan
5 Recruiting Biobank Protocol, Rare Diseases Clinical Research Network
Conditions: Primary Hyperoxaluria;   Dent Disease;   APRT Deficiency;   Cystinuria
Intervention:
6 Recruiting Decision Support for Parents Receiving Information About Child's Rare Disease
Conditions: Disorder of Sex Development;   Intersex Conditions;   Congenital Adrenal Hyperplasia;   Hypospadias
Intervention: Behavioral: Decision Support Tool
7 Recruiting Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions
Condition: Rare Genetic Disorders
Intervention:
8 Recruiting Mitoferrin-1 Expression in Erythropoietic Protoporphyria (Porphyria Rare Disease Clinical Research Consortium (RDCRC))
Condition: Erythropoietic Protoporphyria (EPP)
Intervention:
9 Not yet recruiting BBD Longitudinal Study of Osteogenesis Imperfecta
Condition: Osteogenesis Imperfecta
Intervention:
10 Recruiting The Natural History Study of Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)
Condition: Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)
Intervention:
11 Recruiting Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL)
Conditions: FTLD;   Progressive Supranuclear Palsy (PSP);   Frontotemporal Dementia (FTD);   Corticobasal Degeneration (CBD);   PPA Syndrome;   Behavioral Variant Frontotemporal Dementia (bvFTD);   Semantic Variant Primary Progressive Aphasia (svPPA);   Nonfluent Variant Primary Progressive Aphasia (nfvPPA);   FTD With Amyotrophic Lateral Sclerosis (FTD/ALS);   Amyotrophic Lateral Sclerosis (ALS);   Oligosymptomatic PSP (oPSP);   Corticobasal Syndrome (CBS)
Intervention:
12 Unknown  High Dose Intravenous N-Acetylcysteine Versus Iloprost for Early, Rapidly Progressive Diffuse Systemic Sclerosis
Condition: Scleroderma, Diffuse
Intervention: Drug: N-acetylcysteine (NAC)
13 Recruiting Clinical and Genetic Examination of Usher Syndrome Patients' Cohort in Europe
Condition: Usher Syndrome
Intervention:
14 Recruiting Nephrotic Syndrome Study Network
Conditions: Minimal Change Disease (MCD);   Membranous Nephropathy;   Glomerulosclerosis, Focal Segmental
Intervention:
15 Recruiting Pediatric Vasculitis Initiative
Conditions: Wegeners Granulomatosis (Granulomatosis With Polyangiitis);   Microscopic Polyangiitis;   Churg Strauss Syndrome (Eosinophilic Granulomatosis With Polyangiitis);   Polyarteritis Nodosa;   Takayasu Arteritis;   Primary CNS Vasculitis;   Unclassified Vasculitis
Intervention:
16 Recruiting Canadian Fabry Disease Initiative (CFDI) Enzyme Replacement Therapy (ERT) Study
Condition: Fabry Disease
Interventions: Drug: agalsidase alfa;   Drug: Agalsidase beta
17 Not yet 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
18 Unknown  Pulmonary Hypertension, Quality of Life and Psychosocial Factors
Condition: Pulmonary Hypertension
Intervention: Behavioral: Evaluation of quality of life
19 Not yet recruiting A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With Mucopolysaccharidoses (MPS) 7
Conditions: Sly Syndrome;   MPS VII;   Mucopolysaccharidosis;   Mucopolysaccharidosis VII
Intervention: Drug: UX003
20 Not yet 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

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Indicates status has not been verified in more than two years