Despite the suboptimal imaging conditions, TTE and CMR measurements of LVEF demonstrated high agreement, with a mean difference of 1.6%. Transthoracic echocardiography (TTE) and cardiac magnetic ...

Nintedanib significantly reduced the decline in the forced vital capacity of patients with progressive pulmonary fibrosis (PPF). Treatment with nintedanib significantly slows the progression of ...

An IDEAL-IQ FF threshold of 52.3% in the vastus lateralis accurately identified loss of ambulation with 92.5% sensitivity and 100% specificity. Findings from a recent study published in the Journal of ...

The authors argue that the long-held label of "uniformly lethal" may no longer be appropriate and can inadvertently bias clinical decision-making. Homozygous achondroplasia is traditionally ...

Ambulatory boys with DMD treated with delandistrogene moxeparvovec maintained NSAA scores above baseline at year 3. Sarepta Therapeutics announced positive 3-year functional outcomes from the EMBARK ...

Tafamidis clinically stabilized neuropathy in most patients who developed acquired transthyretin amyloidosis following a domino liver transplantation. Patients who receive livers from individuals with ...

At present, no validated tools or scoring systems have been developed to predict prognosis or clinical course of warm autoimmune hemolytic anemia (AIHA). Several potential prognostic risk factors that ...

Patients with ATTR-CM who experienced outpatient worsening heart failure had a 2.5-fold increased risk of mortality compared with those who did not. Outpatient worsening heart failure is an ...

The results suggest that the mechanism of MTDPS13 is a loss of mitochondrial quantity, not quality. Adenosine monophosphate-activated protein kinase (AMPK) activators restored mitochondrial function ...