In all, 218 proteins showed a significant association with a cardiac index. Four correlated with both LVEF and Ecc, and 42 with both LVEF and LGE. None correlated with all 3 indices. Researchers ...

Comparison with a concurrent cohort of 47 patients with clinically diagnosed wild-type ATTR-CM revealed a striking difference in disease burden. Systematic age- and biopsy-guided screening during ...

The risk of occurrence of the clinical trial's endpoints was lower in patients with PPF treated with 9 mg or 18 mg twice a day compared with those given the placebo. Treatment with nerandomilast at a ...

Exploratory outcomes indicated further benefits, including lower rates of myasthenic crises and hospitalizations, as well as reduced need for rescue therapy. Cemdisiran, a small interfering RNA (siRNA ...

Several genes previously associated with loss of ambulation showed no evidence of interaction with corticosteroid exposure. Genetic modifiers, particularly those in the transforming growth factor β ...

Abby Bronson advocates for those with Becker muscular dystrophy, which can have severe consequences on mobility and quality of life. Larry Luxner, senior correspondent for Rare Disease Advisor, ...

The CAPTIVATE trial met its target of at least 20 responders among the first 40 participants before all 40 completed part A. Dianthus Therapeutics announced an early “go” decision for claseprubart for ...

Concomitant MYD88 p.L265P mutation and MYC copy-number gain in splenic marginal zone lymphoma (SMZL) presenting with warm autoimmune hemolytic anemia (AIHA) may identify a clinically indolent yet ...

Pathogen-reduced plasma, cryoprecipitate reduced (PRPCR), may reduce the risk for transfusion-transmitted infection (TTI) in patients who undergo repeated therapeutic plasma exchange (TPE) for ...

Consolidation or maintenance therapy significantly improved progression-free survival and overall survival. Zanubrutinib, lenalidomide, temozolomide, and rituximab (RLZT) combined with or without high ...

The Reagan-Udall foundation was named for President Ronald Reagan and Congressman Morris “Mo” Udall from Arizona, each of whom had an incurable neurodegenerative disease. The Reagan-Udall Foundation ...

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 ...