A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...

The treatment uses a modified, harmless virus to deliver healthy genes to a patient’s cells to treat genetic diseases. Read ...

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...

The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and ...

The donation from Don and Anne Edwards aims to speed research, treatment and diagnosis for children with rare and genetic ...

Regeneron is adding its name to Tessera Therapeutics’ unique in vivo gene writing program, inking a deal that lets it in on a ...

Ann & Robert H. Lurie Children's Hospital on Wednesday announced what they called a "transformative gift," as investment ...

TripletDGC links disease genes→cell types→diseases via eQTL-scRNA integration, exposing sparse but critical cell drivers and potential comorbidity ...

Fondazione Telethon ETS' Waskyra has become the first gene therapy in the US for patients with Wiskott-Aldrich syndrome (WAS) ...

Don and Anne Edwards have donated $11 million to Ann & Robert H. Lurie Children's Hospital to establish what will be the ...

The donor, who was not identified, was paid to donate as a student beginning in 2005, according to the report.