Patients with MG who took C5 inhibitors saw greater reductions in concomitant OCS and NSIST use than did those who took efgartigimod. Rare Disease Advisor, a trusted source of medical news and feature ...

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 ...

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 ...

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 ...

Ambulatory boys with DMD treated with delandistrogene moxeparvovec maintained North Star Ambulatory Assessment scores above baseline at year 3. Sarepta Therapeutics announced positive 3-year ...

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 ...

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

Fenebrutinib demonstrated noninferiority to ocrelizumab in patients with PPMS, a population with limited treatment options. Genentech has reported that fenebrutinib reduced relapse rates in relapsing ...

Treating individuals with severe thrombocytopenia and frequent bleeding, which is reported in both FNAIT and GNE-related macrothrombocytopenia, is challenging. Fetal and neonatal alloimmune ...