Background: Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Nexiguran ziclumeran (nex-z) is an investigational therapy based on CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats and associated Cas9 endonuclease) targeting the gene encoding transthyretin ().
Methods: In this phase 1, open-label trial, we administered a single intravenous infusion of nex-z to patients with ATTR-CM.
Background: In two phase 3 studies, tenofovir alafenamide (TAF) showed non-inferior efficacy versus tenofovir disoproxil fumarate (TDF), with more favourable renal and bone safety in patients with chronic hepatitis B (CHB).
Aims: Here, we report the studies' final 8-year results.
Methods: CHB patients (hepatitis B e antigen [HBeAg]-negative and HBeAg-positive) were randomised (2:1) to double-blind TAF 25 mg/day or TDF 300 mg/day for up to 3 years, followed by open-label (OL) TAF through year 8.
HBV capsid assembly modulators (CAMs) target the core protein and inhibit pregenomic RNA encapsidation and viral replication. HBV CAMs also interfere with cccDNA formation during de novo infection, which in turn suppresses transcription and production of HBV antigens. In this report, we describe the antiviral activities of AB-836, a potent and highly selective HBV CAM.
View Article and Find Full Text PDFObjective: Persons with vestibular schwannoma suffer from dizziness, imbalance, and decreased function leading to reduced quality of life. Other forms of peripheral vestibular hypofunction show improvements in these signs and symptoms with vestibular rehabilitation; however, the efficacy of this intervention for those with vestibular schwannoma is unknown. Therefore, the aim of this systematic review was to determine the effect of vestibular physical therapy on subjective and objective measures of vestibular symptoms and function in people with vestibular schwannoma.
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