An experimental gene therapy treatment created at UCLA gave a "bubble girl" born with a rare genetic disorder a new life.

“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.

DUBLIN--(BUSINESS WIRE)--The "ADA-SCID - Competitive Landscape and Market Insight, 2018" report has been added to ResearchAndMarkets.com's offering. "ADA-SCID - Competitive Landscape and Market ...

LOS ANGELES, October 29, 2025--(BUSINESS WIRE)--Rarity PBC, a Public Benefit Corporation dedicated to expanding the availability of transformative gene therapies for rare diseases, today announced the ...

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 ...

Following a positive opinion from the EMA, the transfer of marketing authorization of Strimvelis from Orchard Therapeutics has been approved by the European Commission Fondazione Telethon will now be ...

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 ...

ADA-SCID, or severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency, affects about 15 children each year in Europe. A child born with ADA-SCID is unable to fight off everyday ...

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 ...

BOSTON and LONDON, May 11, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced data published in the New England Journal of Medicine (NEJM) ...