Lumasiran: A Review in Primary Hyperoxaluria Type 1
AbstractLumasiran (Oxlumo®), a first-in-class synthetic, double-stranded, ribonucleic acid (RNA) interference molecule targeting glycolate oxidase through silencingHAO1 mRNA, is approved in several countries for patients of any age and stage of kidney function with primary hyperoxaluria type 1 (PH1). Approval was based on results from the phase III ILLUMINATE trials. In the double-blind, placebo-controlled, ILLUMINATE-A trial, subcutaneous lumasiran was significantly more effective than placebo in reducing 24-h urinary oxalate excretion in patients aged ≥ 6 years with PH1; this effect was sustained for ≥ 36 months i...
Source: Drugs - January 22, 2024 Category: Drugs & Pharmacology Source Type: research
Correction to: Nedosiran: First Approval
(Source: Drugs)
Source: Drugs - January 22, 2024 Category: Drugs & Pharmacology Source Type: research
Remdesivir Discontinuation Decisions Based on Thresholds of Aminotransferase in an Observational Registry
ConclusionIn this descriptive study of real-world practice in the USA, clinicians are overall making more conservative treatment decisions than are recommended for consideration in approved drug labeling of discontinuation, with wide variation in the aminotransferase thresholds being used. (Source: Drugs)
Source: Drugs - January 10, 2024 Category: Drugs & Pharmacology Source Type: research
Poly-Agonist Pharmacotherapies for Metabolic Diseases: Hopes and New Challenges
AbstractThe use of glucagon-like peptide-1 (GLP-1) receptor-based multi-agonists in the treatment of type 2 diabetes and obesity holds great promise for improving glycaemic control and weight management. Unimolecular dual and triple agonists targeting multiple gut hormone-related pathways are currently in clinical trials, with recent evidence supporting their efficacy. However, significant knowledge gaps remain regarding the biological mechanisms and potential adverse effects associated with these multi-target agents. The mechanisms underlying the therapeutic efficacy of GLP-1 receptor-based multi-agonists remain somewhat ...
Source: Drugs - December 21, 2023 Category: Drugs & Pharmacology Source Type: research
Spesolimab for the Treatment of Generalized Pustular Psoriasis
This article reviews the evidence thus far on spesolimab, a selective humanized antibody against the IL-36 receptor that was recently licensed in Europe and the United States for the treatment of GPP flares in adults. In phase II, randomized controlled clinical trials, spesolimab led to rapid and effective skin clearance in patients experiencing a GPP flare and demonstrated superiority to placebo in preventing flares for up to 48 weeks with maintenance treatment, with reassuring safety and tolerability profiles. Spesolimab is considered to be a first-in-class medication establishing itself as the standard of care for the t...
Source: Drugs - December 20, 2023 Category: Drugs & Pharmacology Source Type: research
Narlumosbart: First Approval
This article summarizes the milestones in the development of narlumosbart leading to this first approval for the treatment of adults with GCTB. (Source: Drugs)
Source: Drugs - December 19, 2023 Category: Drugs & Pharmacology Source Type: research
Targeting KRAS-Mutated Gastrointestinal Malignancies with Small-Molecule Inhibitors: A New Generation of Breakthrough Therapies
AbstractKirsten rat sarcoma virus (KRAS) is one of the most important and frequently mutated oncogenes in cancer and the mutational prevalence is especially high in many gastrointestinal malignancies, including colorectal cancer and pancreatic ductal adenocarcinoma. The KRAS protein is a small GTPase that functions as an “on/off” switch to activate downstream signaling, mainly through the mitogen-activated protein kinase pathway. KRAS was previously considered undruggable because of biochemical constraints; however, recent breakthroughs have enabled the development of small-molecule inhibitors of KRAS G12C. Thes e drug...
Source: Drugs - December 18, 2023 Category: Drugs & Pharmacology Source Type: research
Comment on: “Recent Human Papillomavirus Vaccination is Associated with a Lower Risk of COVID-19: A US Database Cohort Study”
(Source: Drugs)
Source: Drugs - December 18, 2023 Category: Drugs & Pharmacology Source Type: research
Reply to the Comments on “Recent Human Papillomavirus Vaccination is Associated with a Lower Risk of COVID-19: A US Database Cohort Study”
(Source: Drugs)
Source: Drugs - December 18, 2023 Category: Drugs & Pharmacology Source Type: research
Comment on “Recent Human Papillomavirus Vaccination is Associated with a Lower Risk of COVID-19: A US Database Cohort Study”
(Source: Drugs)
Source: Drugs - December 18, 2023 Category: Drugs & Pharmacology Source Type: research
Targeting KRAS-Mutated Gastrointestinal Malignancies with Small-Molecule Inhibitors: A New Generation of Breakthrough Therapies
AbstractKirsten rat sarcoma virus (KRAS) is one of the most important and frequently mutated oncogenes in cancer and the mutational prevalence is especially high in many gastrointestinal malignancies, including colorectal cancer and pancreatic ductal adenocarcinoma. The KRAS protein is a small GTPase that functions as an “on/off” switch to activate downstream signaling, mainly through the mitogen-activated protein kinase pathway. KRAS was previously considered undruggable because of biochemical constraints; however, recent breakthroughs have enabled the development of small-molecule inhibitors of KRAS G12C. Thes e drug...
Source: Drugs - December 18, 2023 Category: Drugs & Pharmacology Source Type: research
