Genentech, a member of the Roche Group, announced that the primary analysis of the Phase III HAVEN 7 study reinforced the efficacy and safety of Hemlibra® in previously untreated or minimally treated infants with severe hemophilia A without factor VIII inhibitors.
Mirum Pharmaceuticals, Inc. announced that on December 8, 2023, the Compensation Committee of Mirum’s Board of Directors granted inducement awards consisting of non-qualified stock options to purchase 25,750 shares of common stock and 12,880 restricted stock units to five new employees under Mirum’s 2020 Inducement Plan.
Scenic Biotech, a pioneer in the discovery of genetic modifiers developing therapeutics to treat severe diseases, announced positive preclinical data for its lead small molecule QPCTL inhibitor, SC-2882.
bluebird bio, Inc. announced new and updated efficacy, safety and health-related quality of life data from the Phase 1/2 HGB-206 Group C and Phase 3 HGB-210 studies of lovotibeglogene autotemcel gene therapy for sickle cell disease through five years of follow-up.
Transfer RNA’s unique biology could enable a mutation-specific, gene-agnostic—and more cost effective—approach to developing treatments for thousands of rare and ultra-rare diseases.
Opinion: Move Over mRNA; tRNAs Have Even Broader Reach Read More »
Uno de los metales más tóxicos proveniente del entorno natural.
Son reactivos de fase aguda con concentraciones que cambian en respuesta a la inflamación, tanto de forma aguda como crónica
Sito en Villa Domínguez, provincia de Entre Ríos, se emplazó con la llegada de los primeros colonos y hasta se sostuvo por los vecinos durante años. Hoy atiende a cerca de 600 pacientes mensuales. Todo sobre su pasado y presente.
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today positive long-term follow-up data from the pivotal, Phase III KATHERINE study in people with HER2-positive early-stage breast cancer (eBC) who have residual invasive disease following neoadjuvant (before surgery) treatment.
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).
