WVE-006, an advanced candidate for treating alpha-1 antitrypsin deficiency (AATD), is designed to restore functional wild-type AAT protein production and reduce Z-AAT protein aggregation. The agent has the therapeutic potential for disease modification in both lung and liver phenotypes of AATD. Last month, the clinical-stage genetic medicines company Wave Life Sciences hosted a virtual event,…
In patients with a gastrointestinal stromal tumor (GIST), the risk for malignancy can be evaluated with the use of noninvasive radiomics, according to the results of an analysis published in Frontiers in Oncology. A retrospective, multicenter study was conducted among patients with a confirmed histopathologic GIST from 5 medical centers in China. Data from computed…
A rare occurrence of cold agglutinin disease (CAD) that accompanied a COVID-19 infection has been reported in a woman with the hemoglobin E (HbE) heterozygous phenotype. Findings from this case study were recently published in the Annals of Clinical Case Reports. Few case reports of autoimmune hemolytic anemia (AIHA), of which CAD is a type,…
In patients with a resected gastrointestinal stromal tumor (GIST), an elevated preoperative controlling nutritional status (CONUT) score has been shown to be a predictor of recurrence, researchers reported in the Journal of Surgical Oncology. Patients with localized primary GISTs who were treated at the First Affiliated Hospital, Zhejiang University School of Medicine, in Hangzhou, Zhejiang,…
In patients with Duchenne muscular dystrophy (DMD), T1 mapping of the pelvic and thigh muscles can be used as a quantitative biomarker for disease involvement, as well as to evaluate disease severity and motor function. A prospective cohort study on the topic was conducted among a group of Chinese patients with DMD. Results of the…
Plasma complement 3 (C3) and complement 4 (C4) are promising biomarkers for distinguishing neuromyelitis optica spectrum disorder (NMOSD) from myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD), with C3 and C4 linked to blood-brain-barrier (BBB) disruption in individuals with NMOSD. A single-center, retrospective, observational study was conducted among patients recruited from the affiliated brain hospital of Nanjing…
A high prevalence of metabolic disorders, including overweight/obesity and diabetes, has been observed among patients with cholangiocarcinoma (CCA), highlighting the need to further assess patients with the disease who present with suspected laboratory hepatic alterations. A retrospective cohort of patients who were diagnosed with CCA between October 2013 and January 2021 and treated at Instituto…
The use of a novel nanomembrane-based therapeutic plasma exchange (TPE) has been shown to reduce prolonged invasive mechanical ventilation (IMV) during acute exacerbations in patients with myasthenia gravis (MG), according to a new study published in the Journal of Clinical Medicine. A single-center, retrospective, and before-and-after study were conducted among patients with MG with impending…
In patients with myasthenia gravis (MG) and those without the disorder, postsurgical complications following thymoma do not differ, according to the results of a study that was published in Asian Cardiovascular and Thoracic Annals. The researchers sought to explore the impact of MG among patients with thymoma undergoing surgery. A total of 145 consecutive patients…
COVID-19 infection has been shown to increase the risk of new-onset myasthenia gravis (MG), myasthenic crisis, respiratory failure, and mortality rates, all due to the presence of a cytokine storm in these individuals, according to a new study published in the Egyptian Journal of Neurology. Some experimental treatments used in patients with MG can worsen…
