Vutrisiran: A Review in Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis
In conclusion, vutrisiran is an efficacious and generally well-tolerated alternative option for the treatment of polyneuropathy of hATTR amyloidosis, which has the potential advantage of infrequent subcutaneous dosage. (Source: Drugs)
Source: Drugs - September 20, 2023 Category: Drugs & Pharmacology Source Type: research
Alzheimer ’s Disease: Novel Targets and Investigational Drugs for Disease Modification
AbstractNovel agents addressing non-amyloid, non-tau targets in Alzheimer ’s Disease (AD) comprise 70% of the AD drug development pipeline of agents currently in clinical trials. Most of the target processes identified in the Common Alzheimer’s Disease Research Ontology (CADRO) are represented by novel agents in trials. Inflammation and synaptic plasticity/neuroprotec tion are the CADRO categories with the largest number of novel candidate therapies. Within these categories, there are few overlapping targets among the test agents. Additional categories being evaluated include apolipoprotein E\(\varepsilon\) 4 (APOE4) e...
Source: Drugs - September 20, 2023 Category: Drugs & Pharmacology Source Type: research
Escalation Versus Induction/High-Efficacy Treatment Strategies for Relapsing Multiple Sclerosis: Which is Best for Patients?
AbstractAfter more than 2 decades of recommending an escalating strategy for the treatment of most patients with multiple sclerosis, there has recently been considerable interest in the use of high-efficacy therapies in the early stage of the disease. Early intervention with induction/high-efficacy disease-modifying therap y may have the best risk-benefit profile for patients with relapsing-remitting multiple sclerosis who are young and have active disease, numerous focal T2 lesions on spinal and brain magnetic resonance imaging, and no irreversible disability. Although we have no curative treatment, at least seven cl as...
Source: Drugs - September 19, 2023 Category: Drugs & Pharmacology Source Type: research
Pharmacotherapy for Dravet Syndrome: A Systematic Review and Network Meta-Analysis of Randomized Controlled Trials
ConclusionsThere exists first-class evidence that documents the efficacy and tolerability of stiripentol, pharmaceutical-grade cannabidiol, fenfluramine hydrochloride, and soticlestat for the treatment of seizures associated with DS, and allows discussion about the expected outcomes regarding seizure frequency reduction and tolerability profiles. (Source: Drugs)
Source: Drugs - September 11, 2023 Category: Drugs & Pharmacology Source Type: research
Correction to: Remdesivir: A Review in COVID-19
(Source: Drugs)
Source: Drugs - September 4, 2023 Category: Drugs & Pharmacology Source Type: research
Birch Bark Extract: A Review in Epidermolysis Bullosa
AbstractBirch bark extract (Filsuvez®; also known as the developmental name Oleogel-S10), a topical gel consisting of 10% dry birch bark extract and 90% sunflower oil, is the first therapy approved in the EU and UK for the treatment of partial thickness wounds associated with dystrophic and junctional epidermolysis bullosa (EB) in patients aged ≥ 6 months old. In the pivotal double-blind, randomized, vehicle-controlled, phase III EASE trial in patients with EB, the primary endpoint was met, in which birch bark extract relative to control gel significantly increased the proportion of patients with first complete target...
Source: Drugs - September 2, 2023 Category: Drugs & Pharmacology Source Type: research
Rozanolixizumab: First Approval
This article summarizes the milestones in the development of rozanolixizumab leading to this first approval for the treatment of gMG in adults who are anti-AChR or anti-MuSK antibody positive. (Source: Drugs)
Source: Drugs - September 1, 2023 Category: Drugs & Pharmacology Source Type: research
Clinical Outcomes with GLP-1 Receptor Agonists in Patients with Heart Failure: A Systematic Review and Meta-analysis of Randomized Controlled Trials
ConclusionCompared with placebo, GLP-1 RAs may reduce the risk of MACE in patients with HF coexisting with T2DM, with a more significant efficiency of human GLP-1 RAs. More RCTs are needed to assess the cardiovascular benefits of GLP-1 RAs in HF, regardless of T2DM.RegistrationThe protocol for this meta-analysis is registered on PROSPERO [CRD42022357886].Graphical Abstract (Source: Drugs)
Source: Drugs - August 28, 2023 Category: Drugs & Pharmacology Source Type: research
