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Aug 23, 10 03:13AMMembranoproliferative glonerulonephritis (MPGN) is a relatively-rare, immune-mediated kidney disease. All current therapies are inadequate and MPGN frequently leads to kidney failure. This study is a 10 patient trial of the monoclonal antibody rituximab for adult patients with MPGN. Study patients will receive 2 doses of rituximab intravenously on days 1 and 15 and will ...Location: Mayo Clinic Status: Active, not recruiting» Read more
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Aug 23, 10 03:23AMInvestigation of the causes of genetic defects relating to hereditary urologic malignancies will be undertaken. These rare disorders result from inherited or newly arising mutations in genes involved in the development and function of different organ systems. As specific disease syndromes are recognized and the responsible genes identified, mutations in individual fami...Location: National Cancer Institute (NCI) Status: Completed» Read more
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Aug 23, 10 11:36AMApparent mineralocorticoid excess (AME) is a rare inherited disease that can cause severe high blood pressure and low blood potassium in children and adults. It is caused by abnormal hormone metabolism and can be fatal. This study will focus on the genetic basis, natural history, disease progression, and survival of people with AME.Location: Mount Sinai School of Medicine Status: Recruiting» Read more
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Aug 23, 10 01:13PMThis study will examine and test patients with neonatal onset multi-system inflammatory disease (NOMID) to learn more about the cause and course of the disease. It will study the disease signs and symptoms and the possible role of a gene called CIAS1, and it will develop a database to gather information on patients with NOMID in the United States and around the world. ...Location: National Institutes of Health Clinical Center, ... Status: Recruiting» Read more
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Aug 23, 10 01:40PMThe purpose of this study is to investigate the safety and efficacy of isavuconazole in the treatment of invasive fungal infections that are caused by aspergillus and rare fungi.Status: Active, not recruiting» Read more
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Aug 23, 10 01:33PMIdiopathic retroperitoneal fibrosis (IRF) is a rare disease characterised by the presence of a retroperitoneal periaortic fibro-inflammatory tissue which may entrap the ureters and cause renal failure. The treatment of IRF is not well established. Corticosteroids are frequently used, but the anti-estrogen agent tamoxifen has also been reported to be effective in a number...Location: Department of Clinical Medicine Nephrology and ... Status: Active, not recruiting» Read more
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Aug 23, 10 07:18PMPulmonary lymphoangioleiomyomatosis (LAM) is a rare destructive lung disease typically affecting women of childbearing age. Currently, there is no effective therapy for the disease and the prognosis is poor. In order to better study this disease, the National Heart, Lung, and Blood Institute (NHLBI) has developed a registry to keep an official record of patients diagno...Location: National Heart, Lung and Blood Institute (NHLBI) Status: Completed» Read more
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Aug 23, 10 07:20PMThe annual number of cases of clinical malaria worldwide is estimated to be 300-500 million leading to 1.5 million deaths. Delayed care and frequent drug resistance of Plasmodium falciparum (Pf), the most frequent form of malaria, is responsible for these deaths. Each year, 5000-8000 travellers return to France with malaria, 4/5 from Africa and with Pf. Clinical featur...Location: Hopital BICHAT CLAUDE BERNARD Status: Recruiting» Read more
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Aug 23, 10 05:58AMGastroentero-pancreatic neuroendocrine tumours (GEP-NETs) are regarded as a fairly rare disease. They are derived from the neuroendocrine system of the gastrointestinal tract and the pancreas and share common clinical features. So far, there is still uncertainty about the cell biology and mechanistic regulation of these tumours. Therefore targeted treatment is limited an...Location: Div. of Endocrinology, Diabetes and Clinical Nu... Status: Recruiting» Read more
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Aug 23, 10 10:31PMThe purpose of this natural history study is to understand more about the progression of infantile Krabbe disease, a very rare genetic disease. There is very little published longitudinal data with only anecdotal cases. This natural history study will be important in understanding the effect of future therapies that are presently in the preclinical phase.Location: University of North Carolina at Chapel Hill Status: Recruiting» Read more
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