Gene Therapy Being Assessed for Treatment of X-Linked Severe Combined Immunodeficiency
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Gene Therapy Being Assessed for Treatment of X-Linked Severe Combined Immunodeficiency

  Mustang Bio, a biopharmaceutical company, has been working with St. Jude Children's Research Hospital to develop MB-107, a lentiviral gene therapy to treat X-linked severe combined immunodeficiency. Mustang Bio…

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Experimental Myelodysplastic Syndrome Treatment Receives Breakthrough Therapy Designation

  A drug created by Agios Pharmaceuticals, TIBSOVO, was recently granted the Breakthrough Therapy designation by the FDA for the treatment of relapsed or refractory myelodysplastic syndrome (MDS) with an…

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Researchers Identify Method to Help Detect Pulmonary Arterial Hypertension in Systemic Sclerosis Patients
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Researchers Identify Method to Help Detect Pulmonary Arterial Hypertension in Systemic Sclerosis Patients

According to a story from Pulmonary Hypertension News, a recent study has determined that cardiopulmonary exercise testing can be an effective method to help identify systemic sclerosis patients that are…

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27th UEG Week: Anti-TNF-α Therapy as a Treatment for Inflammatory Bowel Disease
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27th UEG Week: Anti-TNF-α Therapy as a Treatment for Inflammatory Bowel Disease

  The 27th United European Gastroenterology Week took place in Barcelona, Spain in October, at which some of the leading experts in gastroenterology spoke about their field. One of the…

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Scientists Find That Rubella and Certain Cancers Have the Same Mutation Mechanisms
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Scientists Find That Rubella and Certain Cancers Have the Same Mutation Mechanisms

By Danielle Bradshaw from In The Cloud Copy Scientific teams at the Centers for Disease Control (CDC), the National Institute of Environmental Health Sciences (NIEHS), and other research centers have…

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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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The Early Results of This Gene Therapy Trial for Phenylketonuria Look Encouraging
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The Early Results of This Gene Therapy Trial for Phenylketonuria Look Encouraging

According to a story from globenewswire.com, the genetic medicines company Homology Medicines, Inc., has recently released promising early data from its phase 1/2 clinical trial. This clinical trial is testing…

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