More than one-third of the elevated serum proteins reflected muscle fiber damage and leakage into the bloodstream. A large, reproducible set of blood-based protein biomarkers can reliably distinguish ...

Almost all (97%) of the patients with positive planar imaging and positive single-photon emission computed tomography findings were diagnosed with ATTR-CM. Single-photon emission computed tomography ...

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 majority (79%) of cardiac amyloidosis cases were responsible for sudden cardiac death. Cardiac amyloidosis can be associated with sudden cardiac death and is often undiagnosed during a person’s ...

More than half (56.3%) of the patients with PBC treated with seladelpar as a second-line therapy achieved a biochemical response. The efficacy of seladelpar as a second-line therapy in patients with ...

Statin treatment reduces the expression of TOMM40 and TOMM22, thereby impairing mitochondrial function. Statins effectively lower plasma low-density lipoprotein cholesterol levels, but they also ...

The treatment could potentially help patients reduce the use of glucocorticoids and immunosuppressants, the authors of the study said. The safety and efficacy of efgartigimod for the treatment of ...

Coramitug is well tolerated and leads to a statistically significant reduction in N-terminal pro-brain type natriuretic peptide (NT-proBNP)—a marker of disease progression in transthyretin-mediated ...

The researchers reported that Scheuer stage score increased significantly with histological stage and GLOBE score. The GLOBE score of patients with primary biliary cholangitis (PBC) before treatment ...

Daily dosing may reduce the metabolic drop-off that occurs prior to each weekly augmentation; therefore, alvelestat represents a potential oral disease-modifying treatment for AATD. Alvelestat ...

The overall response rate of transfusion-dependent patients was 56% while that of transfusion-independent patients was 46%. Momelotinib treatment leads to clear clinical benefits in patients with ...

Professor Aya Abrahamov and two Palestinian women with Gaucher disease share a light moment at Shaare Zedek Medical Center in Jerusalem, Israel (Photo by Ari Zimran) Shaare Zedek’s Gaucher unit is the ...