Claseprubart incorporates YTE half-life extension technology, which is designed to enable infrequent subcutaneous self-administration. A phase 3 study is underway to evaluate the investigational ...

Researchers advanced the therapy with an AI platform that uses machine learning models trained on patient endotyping, multiomics, biomarker, and clinical data. The US Food and Drug Administration (FDA ...

The most important of the tested treatment benefits was reducing the risk of death, while the least important was the ability to easily take a bath or shower. People living with transthyretin-mediated ...

The mixed vascular-fibrotic group demonstrated the shortest median progression-free survival (36 months) and the highest independent risk of disease progression. Researchers proposed a new ...

The main phenotype across countries was ATTR polyneuropathy, affecting between 51.3% and 63.7% of patients. The clinical manifestations and outcomes in hereditary transthyretin amyloidosis (ATTR) are ...

Julian Beach, MHRA executive director, said the agency had assessed the evidence and was “satisfied it meets our standards for safety, quality, and effectiveness.” The UK Medicines and Healthcare ...

(Getty Images/Halfpoint Images) ATTR-CM and heart failure with preserved ejection fraction (HFpEF) caused by other factors tend to present in similar ways, research shows. Transthyretin-mediated ...

In a pooled analysis of 48 emulated trials, the mortality risk was 69.7% lower among individuals who received an exon-skipping therapy plus glucocorticoids than among those who received ...