by Peter Ciszewski | Feb 8, 2019
Manuel Litchman, MD, President, Chief Executive Officer at Mustang Bio discusses his company’s X-linked severe combined immunodeficiency (SCID) program. X-linked SCID is an inherited disorder of the immune system that occurs almost exclusively in males....
by Peter Ciszewski | Feb 7, 2019
The U.S. Food and Drug Administration (FDA) approved Cablivi (caplacizumab-yhdp) injection, the first therapy specifically indicated, in combination with plasma exchange and immunosuppressive therapy, for the treatment of adult patients with acquired thrombotic...
by Peter Ciszewski | Feb 6, 2019
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...
by Peter Ciszewski | Feb 5, 2019
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),...
by Peter Ciszewski | Feb 4, 2019
Other Names: aHUS; Atypical HUS; HUS, atypical Atypical hemolytic uremic syndrome (aHUS) is a disease that causes abnormal blood clots to form in small blood vessels in the kidneys. These clots can cause serious medical problems if they restrict or block blood flow,...
