Using Genomics to Better Understand Rare Diseases

  Daniel MacArthur, PhD is an Institute Member at the Broad Institute of MIT and Harvard, and co-lead of the Broad’s Center for Mendelian Genomics and Rare Genomes Project. Dr. MacArthur is a group leader in the Analytic and Translational Genetics Unit at...

Hereditary ATTR Amyloidosis Overview

  Pushkal Garg, MD, Chief Medical Officer at Alnylam discusses hereditary ATTR (hATTR) amyloidosis, a rare condition that affects an estimated 50,000 people worldwide. Symptoms can affect multiple parts of the body, including the nervous (nerve), cardiac (heart),...

Living with Charcot-Marie-Tooth Disease

Charcot-Marie-Tooth disease (CMT) is one of the most common inherited neurological disorders, affecting approximately 1 in 2,500 people in the United States. The disease is named for the three physicians who first identified it in 1886 – Jean-Martin Charcot and...
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