Ultomiris (ravulizumab) and Soliris (eculizumab), both developed by global biopharmaceutical leader AstraZeneca, are both effective treatments for paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Yet this efficacy…
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.…
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematopoietic stem cell disorder in which blood cells break apart prematurely. In an effort to bring more effective and targeted treatments…
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…
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…
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…
Currently, C5 inhibitors such as eculizumab and ravulizumab are considered the standard-of-care for people living with paroxysmal nocturnal hemoglobinuria (PNH). Unfortunately, not all patients respond well to these therapies.…
According to a news release from biotechnology company Kira Pharmaceuticals, its therapy KP104 earned Orphan Drug designation in the United States. KP104 is in development as a therapeutic option…
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In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare or life-threatening conditions. A “rare” condition is defined as…
This year, the European Hematology Association (EHA) held its Hybrid Conference starting on June 15; if registered for the conference, you may access the Congress Platform until August 15, 2022.…
One thing is sure: 29-year-old Aimee Read was always a fighter. At just two years old, Aimee was diagnosed with leukemia. After two years of chemotherapy and six months…
PNH Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease where blood cells break apart and cause the contents to circulate freely within the bloodstream. This condition is caused by a…
In a late March 2022 news release from biopharmaceutical company CANbridge Pharmaceuticals, Inc. ("CANbridge"), the company noted that the first patient was dosed in a Phase 1b/2 clinical trial evaluating…
According to a recent article, Apellis Pharmaceuticals and Swedish Orphan Biovitrum AB recently announced the European Commission (EU) has approved Aspaveli to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). Paroxysmal…
According to a recent article, the FDA has given approval for Apellis Pharmaceutical’s Inc.’s Empaveli to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). Paroxysmal Nocturnal Hemoglobinuria Paroxysmal nocturnal hemoglobinuria (PNH)…
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…
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…
According to a press release from July 15, 2021, the Chinese NMPA recently approved an Investigational New Drug (IND) application submitted for CAN106, a potential therapeutic option for patients with…
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…
As of June 7, 2021, an injectable treatment is now available for patients aged 1 month+ with paroxysmal nocturnal hemoglobinuria. The FDA recently approved Ultomiris (ravulizumab-cwvz) for these patients. Previously,…
According to FirstWord Pharma, patients with paroxysmal nocturnal hemoglobinuria (PNH) in the United Kingdom (UK) may soon have a more effective and efficient, and less invasive, treatment option. Currently, the…
Have you ever heard of Breakthrough Therapy designation? This designation is granted by the FDA for therapies designed to treat rare, serious, or life-threatening conditions. In mid-December 2020, PMLive…
by Lauren Taylor from In The Cloud Copy Paroxysmal nocturnal hemoglobinuria (PNH) is a very rare disease of the blood in which the affected individuals red blood cells break apart.…
According to a story from Market Watch, the biopharmaceutical company Alexion Pharmaceuticals, Inc. has recently announced that the US Food and Drug Administration (FDA) has approved a new formulation of…
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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