The First Patient in China to be Accepted Under its 2019 Compassionate Use Policy

Beijing, China: According to an article in a sister publication of Sixth Tone, a woman living in China who has been treated during the past fourteen years for paroxysmal nocturnal…

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BCX9930 Shows Promising Results for Patients with Previously Untreated PNH
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BCX9930 Shows Promising Results for Patients with Previously Untreated PNH

On September 30, 2020, BioCryst Pharmaceuticals ("BioCryst") announced promising data on their drug candidate BCX9930. The therapy, an orally administered Factor D inhibitor, is designed to treat patients with previously…

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Pegcetacoplan for Paroxysmal Nocturnal Hemoglobinuria: Marketing Applications Submitted
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Pegcetacoplan for Paroxysmal Nocturnal Hemoglobinuria: Marketing Applications Submitted

  Recently, biopharmaceutical company Apellis Pharmaceuticals announced submission of a New Drug Application (NDA) and Marketing Authorization Application (MAA) for pegcetacoplan. If approved, these applications will allow for patients with…

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NDA to be Submitted for Potential Paroxysmal Nocturnal Hemoglobinuria Treatment
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NDA to be Submitted for Potential Paroxysmal Nocturnal Hemoglobinuria Treatment

According to a story from globenewswire.com, the biopharmaceutical company Apellis Pharmaceuticals, Inc. has announced that it is currently making plans to submit a New Drug Application (NDA) to the US…

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Former UFC Athlete Joe Ellenberger’s Toughest Opponent: Paroxysmal Nocturnal Hemoglobinuria

The UFC has some tough fighters in it. But 35-year-old Joe Ellenberger never thought that his most difficult opponent would be within himself. That is, his paroxysmal nocturnal hemoglobinuria (PNH),…

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Experimental Drug Proves Superior to Eculizumab as a Treatment for Paroxysmal Nocturnal Hemoglobinuria
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Experimental Drug Proves Superior to Eculizumab as a Treatment for Paroxysmal Nocturnal Hemoglobinuria

According to a story from BioSpace, the biopharmaceutical company Apellis Pharmaceuticals, Inc., has recently announced the results of its phase 3 clinical trial, which was testing the company's investigational drug…

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Study: Pozelimab Shows Positive Results in the Treatment of Paroxysmal Nocturnal Hemoglobinuria
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Study: Pozelimab Shows Positive Results in the Treatment of Paroxysmal Nocturnal Hemoglobinuria

  Regeneron Pharmaceuticals has recently announced the results of Phase 2 of their study of pozelimab, which is an experimental treatment for paroxysmal nocturnal hemoglobinuria. Current therapies for this condition…

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Possible Treatment for Paroxysmal Nocturnal Hemoglobinuria Recommended for Marketing Approval in the EU
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Possible Treatment for Paroxysmal Nocturnal Hemoglobinuria Recommended for Marketing Approval in the EU

According to a story from Market Screener, the biopharmaceutical company Alexion Pharmaceuticals has recently announced some encouraging news regarding the status for ravulizumab (marketed as ULTOMIRIS) in the EU. The…

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A Treatment for Paroxysmal Nocturnal Hemoglobinuria Was Accepted for Priority Review

According to a story from Morningstar, the company Alexion Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) has agreed to review their Biologics Licensing Application for their…

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Possible Treatment for Paroxysmal Nocturnal Hemoglobinuria Submitted for FDA Approval
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Possible Treatment for Paroxysmal Nocturnal Hemoglobinuria Submitted for FDA Approval

According to a story from Business Wire, the company Alexion Pharmaceuticals recently announced that it has submitted a Biologics License Application (BLA) to the FDA for the approval of one…

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Recent Study Results Reveal New Possibilities in Treating Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH causes the immune system to destroy red blood cells. Source: pixabay.com

Recent Study Results Reveal New Possibilities in Treating Paroxysmal Nocturnal Hemoglobinuria (PNH)

On December 11, Alexion Pharmaceuticals, Inc. released dose-ranging data from a recent clinical study involving treatment with ALXN1210, which is an investigational long-acting C5 complement inhibitor meant to treat sufferers…

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