Ruxoprubart Granted Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH)
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Ruxoprubart Granted Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH)

Developing novel therapeutics for rare diseases is increasingly important. Understanding the unmet needs of rare disease communities and providing effective, accessible therapies can significantly improve the lives of those affected.…

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Study: Risk Factors for Paroxysmal Nocturnal Hemoglobinuria Thromboembolic Events
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Study: Risk Factors for Paroxysmal Nocturnal Hemoglobinuria Thromboembolic Events

Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…

Continue Reading Study: Risk Factors for Paroxysmal Nocturnal Hemoglobinuria Thromboembolic Events
FACIT-Fatigue Scale: A 5-Point Change is Important for Paroxysmal Nocturnal Hemoglobinuria
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FACIT-Fatigue Scale: A 5-Point Change is Important for Paroxysmal Nocturnal Hemoglobinuria

Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…

Continue Reading FACIT-Fatigue Scale: A 5-Point Change is Important for Paroxysmal Nocturnal Hemoglobinuria
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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