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 ...

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

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

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