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Rare Disease Report®

The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.

Alnylam plans to submit a NDA for the orphan drug used to treat hATTR with amyloidosis in adults.

Vutrisiran Shows Promise Treating hATTR in Topline Results

New data from an ongoing trial showed patients achieved MRD negative status after a vaccine regimen—and have maintained status over a year.

Vaccine Could Provide Long-Term Acute Myeloid Leukemia Remission

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

The HCPLive amyloidosis page is a resource for medical news and expert insights on the rare disease. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for amyloid buildup, and more.