Telethon Foundation is seeking approval for a gene therapy to treat the rare disease Wiskott-Aldrich syndrome.

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Fondazione Telethon, an Italian biomedical charity focused on rare genetic diseases, has submitted a Marketing Authorization ...

PTC Therapeutic's Upstaza gene therapy for patients with genetic disorder ... "bubble boy" disease adenosine deaminase deficiency (ADA-SCID) – originally developed by GlaxoSmithKline ...