Tag: rare diseases

Recommendations Presented for Rare Autoinflammatory Diseases

As researchers learn more about the genetic etiology of immunopathology, they have been able to more clearly understand rare but debilitating autoinflammatory conditions in ways...

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...

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...

Ophthalmologists Called to Catch Cancers Earlier

Significant advances in the management of eye cancer mean that early referrals to subspecialists are crucial when ophthalmologists suspect disease in their patients, two leaders...