Articles

Podcasts

Videos

Rare Disease Report®

The RareWear program is designed to bridge medical device companies to rare disease patient applicants, seeking better disease and treatment monitoring.

Rare Genomics Institute Launches Free Rare Disease Device Program

New research suggests proposals aimed at reducing out of pocket costs may harm minimally-funded drug developers.

Medicare Part D Bills Could Harm Rare Disease Drug Development

The new indication will allow the longtime ALS therapy to be used among the 85% of patients required to use PEG due to dysphagia.

FDA Expands ALS Oral Drug Riluzole to Feeding Tube Use

A fourth-year cardiology medical student shares his perspectives from the audience at the American Heart Association Scientific Sessions.

Notes From Rounds: AHA Day Two

A new gene expression-tracking study, conducted under a novel computation, could provide investigators material to make diagnosis and therapeutic breakthroughs.

ALS Gene Expression Atlas Could Provide New Disease Insights

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

Top 5 Rare Disease News of the Week&mdash;August 5, 2018

The FDA has approved patisiran infusion for the treatment of peripheral nerve disease caused by hereditary transthyretin-mediated amyloidosis in adult patients.

FDA Approves First Treatment for Polyneuropathy Caused by hATTR

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.

Geographic & Racial Disparities Found in Reported US Amyloidosis Mortality

hATTR compass program will empower people with accurate genetic information so they can make informed decisions about their healthcare.

New Program May Help Cut Time to Diagnosis for hATTR Patients

Ray Comenzo, MD, highlights promising ANDROMEDA phase 3 trial, which evaluated the efficacy of subcutaneous daratumumab and cyclophosphamide, bortezomib, and dexamethasone (CyBorD) as a combination therapy for newly diagnosed light chain (AL) amyloidosis patients.