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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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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Paroxysmal Nocturnal Hemoglobinuria Investigative Therapy Moving to Phase 3 Trial in 2020

The EMA has just announced that Danicopan has been accepted into its PRIority MEdicines (PRIME) program. Danicopan is being developed for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood condition. PRIME…

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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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The Aplastic Anemia and MDS International Foundation’s Living with Aplastic Anemia, MDS, PNH Conference

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The 2019 Living With Aplastic Anemia, MDS, PNH Patient and Family Conference This unique conference, which will be held in Albuquerque, New Mexico is an exceptional opportunity for patients with aplastic…

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Aplastic Anemia and MDS International Foundation’s Living with Aplastic Anemia, MDS, or PNH Patient Conference in Miami

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Living with Aplastic Anemia, MDS, or PNH Patient Conference Join us and connect with patients, families, and caregivers who are living with aplastic anemia, PNH, MDS, and AML.  Meet and hear top medical experts…

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Aplastic Anemia and MDS International Foundation’s Living with Aplastic Anemia, MDS, or PNH Patient Conference in Los Angeles

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Living with Aplastic Anemia, MDS, or PNH Patient Conference Join us and connect with patients, families, and caregivers who are living with aplastic anemia, PNH, MDS, and AML.  Meet and hear top medical experts…

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Aplastic Anemia and MDS International Foundation’s Living with Aplastic Anemia, MDS, or PNH Patient Conference in Providence, RI

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Living With Aplastic Anemia, MDS, or PNH Join us and connect with patients, families, and caregivers who are living with aplastic anemia, PNH, MDS, and AML.  Meet and hear top medical experts discuss…

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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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Aplastic Anemia and MDS International Foundation’s Living with Aplastic Anemia, MDS, and PNH Conference

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A patient and family conference for people with aplastic anemia, myelodysplastic syndromes, paroxysmal nocturnal hemoglobinuria, and acute myeloid leukemia. Nurses, caregivers, or other health professionals are also encouraged to attend. Learn more…

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