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.
Wedbush upgrades Sarepta Therapeutics, citing overdone selloff and potential upside as FDA could re-approval Elevidys for non ...
For the scientific enterprise, 2025 was marked by setbacks and challenges. But scientists are no strangers to adversity—the ability to overcome obstacles is built into our training. In turn, research ...
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