“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 ...

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 ...

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 ...

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

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.

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 ...

A long-term follow-up study tracking a group of children who received experimental gene therapy as part of an early-stage clinical trial is reporting the treatment is safe and effective up to 10 years ...

The Nachem family. When Eliana, third from right, was diagnosed with a severe immune condition as an infant, the family had to rehome their pets. (Courtesy Caroline Nachem ) Two months after she was ...