“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.

The alternative text for this image may have been generated using AI. During the sixth month of PEG-ADA discontinuation, with the patient in good clinical status, a routine examination of the fundus ...

"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 ...

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 ...

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 ...

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 ...

ADA-SCID is an ultra-rare, inherited genetic disorder, caused by a deficiency in the ADA enzyme that is fatal if left untreated. Patients affected by ADA-SCID have compromised immune systems that ...

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 ...

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) ...

Leadiant Biosciences, Inc. today announced that the Food and Drug Administration (FDA) has granted approval to Revcovi ™ (elapegademase-lvlr) injection in the U.S. Revcovi is a new enzyme replacement ...

Adenosine deaminase (ADA) deficiency leads to build up of toxic metabolites that causes severe combined immunodeficiency (SCID) and a systemic metabolic defect. ADA–SCID is an ultra-rare condition ...

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.