Alexion Pharmaceuticals, Inc.’s Managing Your PNH Webinar

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Managing Your PNH Connect. Learn. Take Action. December 9, 2021, 6:30 PM Connecting patients and caregivers in the paroxysmal nocturnal hemoglobinuria (PNH) community. To register, click here. Acknowledgment: This event…

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Alexion Pharmaceuticals, Inc.’s Managing Your PNH Webinar

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Managing Your PNH Connect. Learn. Take Action. November 9, 2021, 7:30 PM Connecting patients and caregivers in the paroxysmal nocturnal hemoglobinuria (PNH) community. To register, click here. Acknowledgment: This event…

Continue Reading Alexion Pharmaceuticals, Inc.’s Managing Your PNH Webinar
Study Shows Eculizumab can Improve Outcomes and Quality of Life for Paroxysmal Nocturnal Hemoglobinuria Patients
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Study Shows Eculizumab can Improve Outcomes and Quality of Life for Paroxysmal Nocturnal Hemoglobinuria Patients

Paroxysmal nocturnal hemoglobinuria (PNH) occurs from a mutated PIG-Agene. This causes blood cells to be produced which don't have surface proteins. As such, the cells aren't protected adequately from the complement…

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Retrospective Study Investigates Safety of Pregnancy in Paroxysmal Nocturnal Hemoglobinuria

A recent study documenting cases of pregnancy in individuals living with paroxysmal nocturnal hemoglobinuria (PNH) was published in the Saudi Journal of Medicine & Medical Sciences. Previously, research on pregnancy with…

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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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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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