A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would ...
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 ...
An experimental gene therapy treatment created at UCLA gave a "bubble girl" born with a rare genetic disorder a new life.
The donation from Don and Anne Edwards aims to speed research, treatment and diagnosis for children with rare and genetic ...
The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and ...
The rate of severe infections decreased from 2 infections to 0.2 infections per PYO after treatment with etuvetidigene autotemcel.
Waskyra, developed by Fondazione Telethon, is the first gene therapy that the FDA has approved from a nonprofit applicant.
FDA approves 1st gene therapy for Wiskott-Aldrich syndrome, offering a new treatment for patients lacking matched stem cell donors.
Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...
EpilepsyGTx, a biotechnology company focused on research and development of cutting-edge gene therapies to treat refractory epilepsy, today announced it has raised $33 million in a Series A financing ...
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