FDA Grants Orphan Drug Designation for DCR-A1AT for Treatment of Alpha-1 Antitrypsin Deficiency
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FDA Grants Orphan Drug Designation for DCR-A1AT for Treatment of Alpha-1 Antitrypsin Deficiency

Dicerna Pharmaceuticals works in ribonucleic interference (RNAi) therapies as treatments of rare diseases. They have recently been granted the orphan drug designation by the European Commission for their drug, DCR-A1AT.…

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Study: Inflammatory Bowel Disease Makes Young People Five Times More Likely to Develop Infections
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Study: Inflammatory Bowel Disease Makes Young People Five Times More Likely to Develop Infections

  A new study has found that young people who have inflammatory bowel disease (IBD) are five times more likely to develop an infection than the general population. They also…

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Study: Avapritinib Shows No Adverse Effects in Phase II Trial for Systematic Mastocytosis
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Study: Avapritinib Shows No Adverse Effects in Phase II Trial for Systematic Mastocytosis

  A PIONEER trial has been conducted in order to evaluate the effects of avapritinib, a drug meant to treat systematic mastocytosis. Researchers want to find a better treatment for…

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Zotiraciclib, a New Treatment for Glioma, is Granted Orphan Drug Designation
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Zotiraciclib, a New Treatment for Glioma, is Granted Orphan Drug Designation

  The European Organization for Research and Treatment of Cancer (EORTC) has been evaluating zotiraciclib, which is a new treatment for glioma. This drug has recently been granted the Orphan…

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FDA Grants Orphan Drug Designation for Treatment of Pancreatic Neuroendocrine Tumors
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FDA Grants Orphan Drug Designation for Treatment of Pancreatic Neuroendocrine Tumors

  Surufatinib was recently granted Orphan Drug designation by the FDA. This drug was made for the treatment of pancreatic neuroendocrine tumors (PanNETs). It was created by Hutchison China MediTech…

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New Center for Research of Primary Sclerosing Cholangitis Created in Boston
Fall is a GREAT time of year to visit Boston--and you feel AWESOME about helping to raise funds for PI! [Source: pixabay.com]

New Center for Research of Primary Sclerosing Cholangitis Created in Boston

Copyscape score: 2% Brigham and Women's is launching the nation's first ever center to conduct research on primary sclerosing cholangitis, a rare liver disease that affects about 30,000 people in…

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Study: Idursulfase Therapy is a Safe Treatment for Hunter Syndrome (MPS II)
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Study: Idursulfase Therapy is a Safe Treatment for Hunter Syndrome (MPS II)

  A recent meta-analyses of clinical studies and case reports that examined the safety of idursulfase therapy, which is an enzyme therapy, has shown that this form of treatment is…

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