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…
Managing Your PNH Connect. Learn. Take Action. Live Webinar Wednesday, December 9, 2020 Alexion invites patients, families, and caregivers living with paroxysmal nocturnal hemoglobinuria (PNH) to an educational program for…
Managing Your PNH Connect. Learn. Take Action. Live Webinar Thursday, October 29, 2020 Alexion invites patients, families, and caregivers living with paroxysmal nocturnal hemoglobinuria (PNH) to an educational program for…
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…
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…
As reported in BioSpace; today, Biocryst Pharmaceuticals has been given a boost to further their work to explore the merits of a potential oral treatment for PNH. The U.S. FDA…
On August 31, 2020, biopharmaceutical company Omeros Corporation announced that it will soon move forward with clinical trials for OMS906. This decision follows clearance of an Investigational New Drug Application…
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…
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…
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…
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…
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…
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…
What's New in Aplastic Anemia and PNH? Important Findings from ASH 2018 This online webinar with Dr. Bhumika Patel will discuss the latest aplastic anemia and PNH research presented at…
Big news out of the FDA last week! Alexion Pharmaceuticals got approval for ALXN1210 for paroxysmal nocturnal hemoglobinuria, or PNH, which will now be sold as Ultomiris, while Stemline got approval…
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…
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…
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…
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…
The 7th Seattle Patient Conference: Aplastic Anemia, MDS, and PNH A patient and family conference for people with aplastic anemia, myelodysplastic syndromes, paroxysmal nocturnal hemoglobinuria, and acute myeloid leukemia. Nurses, caregivers, or…
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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