January 11, 2021
Article
Alnylam plans to submit a NDA for the orphan drug used to treat hATTR with amyloidosis in adults.
December 05, 2020
New data from an ongoing trial showed patients achieved MRD negative status after a vaccine regimen—and have maintained status over a year.
February 29, 2020
The RareWear program is designed to bridge medical device companies to rare disease patient applicants, seeking better disease and treatment monitoring.
New research suggests proposals aimed at reducing out of pocket costs may harm minimally-funded drug developers.
December 14, 2019
The new indication will allow the longtime ALS therapy to be used among the 85% of patients required to use PEG due to dysphagia.
November 19, 2019
A fourth-year cardiology medical student shares his perspectives from the audience at the American Heart Association Scientific Sessions.
April 05, 2019
A new gene expression-tracking study, conducted under a novel computation, could provide investigators material to make diagnosis and therapeutic breakthroughs.
August 11, 2018
Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.
August 10, 2018
The FDA has approved patisiran infusion for the treatment of peripheral nerve disease caused by hereditary transthyretin-mediated amyloidosis in adult patients.
August 07, 2018
The lack of higher reported mortality rates in states with a greater proportion of black residents suggests underdiagnosis of amyloidosis, including cardiac forms of the disease, in many areas of the United States.
