A single dose of Homology Medicines’ gene therapy candidate HMI-104 led to the sustained production of a therapeutic antibody to treat paroxysmal nocturnal hemoglobinuria (PNH), preclinical studies suggest. “Given the severity of PNH, which is a rare, acquired blood disorder, and the unmet need associated with chronically administered…
European regulators have recommended approving Epysqli, a Soliris (eculizumab) biosimilar, to treat children and adults with paroxysmal nocturnal hemoglobinuria (PNH). The recommendation came from the Committee for Medicinal Products for Human Use (CHMP) and will serve as the basis for a final approval by the European Commission. Epysqli…
Most people with paroxysmal nocturnal hemoglobinuria (PNH) benefited from Soliris (eculizumab) therapy when they adhered to strict treatment guidelines, a real-world Dutch study showed. Because most had an incomplete response, new therapies are needed to improve real-world outcomes, however, the study’s researchers suggested, and emphasized that their data could…
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