Dustin Vidrine's sight is disappearing — shrinking, really. He has retinitis pigmentosa, a rare, inherited eye disease that runs in his family. By the time he was in his 20s, he noticed that his ...

The FDA has approved Fondazione Telethon ETS’s etuvetidigene autotemcel (Waskyra) for Wiskott–Aldrich syndrome (WAS). Etuvetidigene autotemcel is the first gene therapy to be approved for this rare ...