Rare Genomics Institute Launches Free Rare Disease Device Program

February 29, 2020


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

Medicare Part D Bills Could Harm Rare Disease Drug Development

February 29, 2020


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

FDA Expands ALS Oral Drug Riluzole to Feeding Tube Use

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.

Notes From Rounds: AHA Day Two

November 19, 2019


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

ALS Gene Expression Atlas Could Provide New Disease Insights

April 05, 2019


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

Top 5 Rare Disease News of the Week—August 5, 2018

August 11, 2018


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

FDA Approves First Treatment for Polyneuropathy Caused by hATTR

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.

Geographic & Racial Disparities Found in Reported US Amyloidosis Mortality

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.

New Program May Help Cut Time to Diagnosis for hATTR Patients

June 22, 2018


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

Ray Comenzo, MD, Discusses ANDROMEDA Trial in AL Amyloidosis Patients

June 19, 2018


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.