WebPurpose of Study. The purpose of this study is to evaluate the effectiveness and safety of IONIS-FB-LRx, an antisense inhibitor of complement factor B messenger ribonucleic acid (CFB mRNA), and to evaluate the effect of IONIS-FB-LRx on plasma factor B (FB) levels and serum AH50, CH50 activity in participants with primary immunoglobulin A (IgA) … Web9 apr. 2024 · Objective: Evaluate the efficacy and safety of laquinimod in patients with Huntington disease (HD). Background: Laquinimod has been shown to modulate CNS-resident inflammatory pathways involved in the pathology of HD. Design/Methods: LEGATO-HD compared three dose arms (0.5, 1.0, and 1.5 mg once daily) versus placebo in a 52 …
Ionis partner licenses rare kidney disease treatment and will …
Web18 jan. 2024 · Roche has outlined a new clinical path forward for a closely watched Huntington's disease drug ten months after disappointing Phase 3 study results … Web18 jan. 2024 · In December 2024, Roche licensed the investigational medicine from Ionis. The tominersen clinical development program included the following studies: GENERATION HD1: a randomized, multicenter, double-blind, placebo-controlled Phase 3 clinical study evaluating the efficacy and safety of treatment with tominersen in people with manifest … how many princely states in india
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Web26 jun. 2024 · IONIS-HTT Rx is the first therapy in clinical development targeting the cause of HD by reducing the production of the toxic mutant huntingtin protein (mHTT) from the mutated huntingtin gene. “We are encouraged by the safety profile of IONIS-HTT Rx we have observed to date in the completed dosing cohorts in the Phase 1/2a study. Web26 feb. 2024 · The safety and tolerability of IONIS-FB-Lrx will be assessed by determining the incidence and severity of adverse events that are related to treatment with IONIS-FB-Lrx Proportion of patients in each treatment group achieving a >50% reduction of plasma complement factor B [ Time Frame: Up to 74 weeks ] Web10 apr. 2024 · In 2024, Roche acquired Cytokinetics, Inc.'s SMA program, which is a muscle activator drug for SMA. This acquisition is part of Roche's effort to become a leading company in the SMA market. how could i live without you lyrics hillsong