by Peter Ciszewski | Oct 29, 2019
Bobby Gasper, MD Chief Scientific Officer of Orchard Therapeutics talks about his company’s gene therapy in development to treat children with adenosine deaminase severe combined immunodeficiency (ADA-SCID). “These children are born without functional...
by Peter Ciszewski | Oct 25, 2019
Gary Joseph Lelli, MD, of the Department of Ophthalmology at Weill Cornell Medicine talks about thyroid eye disease due to Graves’ disease, including which clinicians are most likely to diagnose this condition and the most common symptoms in this highly...
by Peter Ciszewski | Oct 24, 2019
The U.S. Food and Drug Administration has approved Vertex’s Trikafta (elexacaftor/ tezacaftor/ ivacaftor and ivacaftor) for the treating persons, 12 years and older, with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane...
by Peter Ciszewski | Oct 23, 2019
Kristin Smedley is the President of Curing Retinal Blindness Foundation, a non-profit organization focused on finding treatments and cures for CRB1 retinal disease. Mutation in the CRB1 gene can lead to two rare conditions – retinitis pigmentosa and Leber...
by Peter Ciszewski | Oct 22, 2019
The U.S. Food and Drug Adminstration (FDA) has approved Ultomiris (ravulizumab-cwvz) to treat adults and children (> 1 month of age) with atypical hemolytic uremic syndrome (aHUS). aHUS is an ultra-rare disease, characterized by inflammation and the formation of...
