Tag: amyloidosis

Did Unrecognized Cardiac Amyloidosis Hinder Recent HFpEF Trials?

Screening for cardiac amyloidosis and excluding patients with cardiac amyloidosis from trials of new therapies for heart failure with preserved ejection fraction (HFpEF) may increase...

Kids in ICU for COVID Are Likely to be Older, Black, Have Asthma

Editor’s note: Find the latest COVID-19 news and guidance in Medscape’s Coronavirus Resource Center. Little has been known about children sick enough with COVID-19 to...

FDA Approves First and Only Treatment for AL Amyloidosis

The first and only treatment for a rare and often fatal blood cell disorder has been approved in the United States. The new indication is...

Positive Phase 3 Trial in hATTR Amyloidosis With Polyneuropathy

The investigational RNAi therapeutic vutrisiran (Alnylam Pharmaceuticals) met its primary and secondary endpoints in a phase 3 study of adults with hereditary transthyretin-mediated amyloidosis (hATTR)...

Low Plasma Transthyretin Linked to Risk of Heart Failure

Low plasma transthyretin, also known as prealbumin, is associated with incident heart failure in the general population, new research suggests. In two prospective, population-based studies...

CDER Chief Reflects on Advances in Rare Diseases

Editor’s note: Find the latest COVID-19 news and guidance in Medscape’s Coronavirus Resource Center. Dr Janet Woodcock Since joining the Food and Drug Administration in...