Concizumab May Be Effective Prophylaxis for Hemophilia A or B With Inhibitors
FRIDAY, Sept. 1, 2023 (HealthDay News) — Patients with hemophilia A or B with inhibitors have a lower annualized bleeding rate with concizumab than with no prophylaxis, according to a phase 3 study published online Aug. 31 in the New England Journal …
Learn MoreLa FDA aprueba la primera terapia genética para la hemofilia A grave
VIERNES, 30 de junio de 2023 (HealthDay News) — El jueves, la Administración de Alimentos y Medicamentos (FDA) de EE. UU. aprobó una costosa terapia genética de una sola dosis para los pacientes con una hemofilia A grave, que es un trastorno heredita…
Learn MoreFDA Approves First Gene Therapy for Severe Hemophilia A
FRIDAY, June 30, 2023 (HealthDay News) — The U.S. Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A. The treatment, Roctavian (valoctocogene roxaparvovec), will cost $2.9 million…
Learn MoreFDA Approves First Gene Therapy for Severe Hemophilia A
FRIDAY, June 30, 2023 (HealthDay News) — The U.S. Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A, a life-threatening hereditary bleeding disorder.The treatment is not cheap: R…
Learn MoreMonthly Injections Might Lower Bleeding in People With Hemophilia
FRIDAY, March 31, 2023 (HealthDay News) — An experimental injectable drug appears effective in reducing bleeds in patients with hemophilia A and B, according to a pair of new clinical trials.Two-thirds of people with treatment-resistant hemophilia wh…
Learn MoreFitusiran Beneficial in Hemophilia A, B With or Without Inhibitors
THURSDAY, March 30, 2023 (HealthDay News) — For males aged 12 years or older with severe hemophilia A or B with or without inhibitors, the annualized bleeding rate is lower with fitusiran prophylaxis, according to two studies published online March 2…
Learn MoreBenefits of Valoctocogene Roxaparvovec Persist in Hemophilia A
FRIDAY, March 3, 2023 (HealthDay News) — For patients with hemophilia A, factor VIII activity and bleeding reduction persist at two years after gene transfer with valoctocogene roxaparvovec, which delivers a B-domain-deleted factor VIII coding sequen…
Learn MoreGene Therapy Beneficial for Patients With Hemophilia B
FRIDAY, March 3, 2023 (HealthDay News) — For patients with hemophilia B, one infusion of adeno-associated virus 5 (AAV5) vector expressing the Padua factor IX variant (etranacogene dezaparvovec) is noninferior and superior to prophylaxis with factor …
Learn MoreOnce-Weekly Efanesoctocog Alfa Beneficial in Severe Hemophilia A
THURSDAY, Jan. 26, 2023 (HealthDay News) — Once weekly efanesoctocog alfa provides superior bleeding prevention to prestudy prophylaxis for patients with severe hemophilia A, according to a study published in the Jan. 26 issue of the New England Jour…
Learn MoreLa FDA aprueba una terapia genética para la hemofilia que cuesta 3.5 millones de dólares
MIÉRCOLES, 23 de noviembre de 2022 (HealthDay News) — Las personas con una forma de hemofilia, un trastorno genético de la sangre, tienen ahora un tratamiento, que se administra una sola vez, que cuesta 3.5 millones de dólares.La Administración de Al…
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