“I remember asking the doctor if my daughter was going to die,” said Chelsea Oakley, mother of Cora Oakley, a newborn who was diagnosed with ADA-SCID when she was just seven days old, in April 2017.

Overall and event-free survival of 100 and 95%, respectively, seen with autologous CD34+ hematopoietic stem-cell lentiviral gene therapy. HealthDay News — Autologous CD34+ hematopoietic stem-cell ...

GAITHERSBURG, Md.--(BUSINESS WIRE)--Leadiant Biosciences, Inc. today announced that the Food and Drug Administration (FDA) has granted approval to Revcovi™ (elapegademase-lvlr) injection in the U.S.

For decades, scientists have been trying to find ways to cure disorders that can be traced back to an error in one gene. One of the first diseases that researchers ...

"Strimvelis returns home." With these words, Alessandro Aiuti, MD, PhD, head of the Pediatric Immunohematology Unit at the Scientific Institute for Research and Healthcare (IRCCS) San Raffaele ...

100% overall survival and ≥95% event-free survival observed at two and three years following one-time treatment withlentiviral HSC gene therapy 50 total participants represent largest published ...

Over a decade ago, UCLA physician-scientists began using a pioneering gene therapy they developed to treat children born with a rare and deadly immune system disorder. They now report that the effects ...

MILAN, BOSTON, and LONDON, Sept. 12, 2023 /PRNewswire/ -- Fondazione Telethon, one of the main Italian biomedical charities, and Orchard Therapeutics, a global gene therapy leader, today announced the ...

An experimental gene therapy developed to treat children born with a rare immunodeficiency disease has demonstrated extraordinary efficacy according to a new long-term follow-up study published in the ...