Fabry Disease: A Biologics License Application for Pegunigalsidase Alfa has Been Submitted to the FDA
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Fabry Disease: A Biologics License Application for Pegunigalsidase Alfa has Been Submitted to the FDA

According to a story from prnewswire.com, the Chiesi Global Rare Diseases and the biopharmaceutical company ProTalix Biotherapeutics have recently announced that they have submitted a Biologics License Application (BLA) for…

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Mouse Model of Gene Therapy is Shown to be Effective Against Hemophilia A
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Mouse Model of Gene Therapy is Shown to be Effective Against Hemophilia A

By Danielle Bradshaw from In The Cloud Copy The cells of hemophilia A patients were taken and genetically modified so that they would create an active clotting factor VIII (or…

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C4 Genes as a Double-Edged Sword: Sex-Based Differences in Lupus, Schizophrenia, and Sjögren’s Syndrome
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C4 Genes as a Double-Edged Sword: Sex-Based Differences in Lupus, Schizophrenia, and Sjögren’s Syndrome

  Women can do anything that men can do, and vice versa. Well, while that sentiment may be true in our everyday life, it isn't quite true on a genetic…

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An App in New Zealand Allows IBD Patients to Communicate with Their Specialists Virtually

IBDsmart Researchers at the University of Otago, led by Michael Shultz, have developed a new app called IBDsmart to help patients with ulcerative colitis or Crohn's disease receive care remotely.…

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Study Identifies Bronchiectasis Risk Factors in Aspirin-Exacerbated Respiratory Disease
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Study Identifies Bronchiectasis Risk Factors in Aspirin-Exacerbated Respiratory Disease

According to a story from Pulmonology Advisor, a recent study has evaluated risk factors for the appearance of bronchiectasis in patients with aspirin-exacerbated respiratory disease (AERD). A small number of…

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A New and Beneficial Tactic for People with Multiple Sclerosis? Mindfulness Training
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A New and Beneficial Tactic for People with Multiple Sclerosis? Mindfulness Training

Being at peace with ourselves and our lives makes daily living so much easier. Of course, sometimes it's easier said than done. But one tactic may help improve comfort, confidence,…

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This Muscular Dystrophy Patient has Spent Her Life Working to Improve the Lives of Others
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This Muscular Dystrophy Patient has Spent Her Life Working to Improve the Lives of Others

Jalee Pelissier lives with muscular dystrophy. When she was just 13 she became an advocate for others with disorders or disabilities like her own. Now 20, her advocacy has only…

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