Alpha-1 Antitrypsin Deficiency Treatment Receives Orphan Drug Designation
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Alpha-1 Antitrypsin Deficiency Treatment Receives Orphan Drug Designation

  In June 2019, Dicerna Pharmaceuticals submitted a clinical trial application for their DCR-A1AT therapy and expects to treat the first patient with A1AD-related liver disease near the end of…

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It Just Makes “Antisense:” Using Genetic Medicine to Treat Rare Diseases
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It Just Makes “Antisense:” Using Genetic Medicine to Treat Rare Diseases

  As previously published in Scientific American, slightly after her first birthday, Emma Larson lost the use of her legs and started experiencing difficulty crawling. She was diagnosed with spinal muscular…

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C. Light Cofounders Discuss Neurotech, Multiple Sclerosis, and Disease Detection
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C. Light Cofounders Discuss Neurotech, Multiple Sclerosis, and Disease Detection

  In an interview with Medgadget, Dr. Christy Sheehy and Dr. Zachary Helft of C. Light Technologies discuss up-and-coming technologies in the diagnosis and treatment of neurological diseases such as…

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Mother of Two Immunocompromised Children Offers Biggest Advice for Surviving COVID-19
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Mother of Two Immunocompromised Children Offers Biggest Advice for Surviving COVID-19

  Mother of two, Mina Manchester, has been navigating raising two children with an immunocompromising disorder for four years. It's this unique experience that makes her feel more prepared and…

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