Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...

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 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 ...

Three‐dimensional imaging revealed a loss of normal microvascular architecture, areas of hyper- and hypovascularization, and microvascular obstruction due to capillary thrombosis.

No ICANS or deaths occurred, and all serious adverse events resolved without sequelae. Cizutamig, a T-cell engager, is generally safe and well tolerated in generalized myasthenia gravis (MG) and other ...

Older age, a history of atrial fibrillation, and carpal tunnel syndrome were associated with a reduced chance of delayed ATTR-CM diagnosis. There are substantial delays from the time of incident heart ...

Results demonstrated substantially worse tolerability in the ATTR-CM subgroup, with 11 out of 17 patients affected by adverse events after starting sacubitril/valsartan compared with 212 out of 978 ...

Overall survival trended favorably with luspatercept across subgroups with extended follow-up. Long-term data from the phase 3 COMMANDS trial (NCT03682536) indicate that luspatercept yields sustained ...