Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by ...
A blood stem cell gene therapy co-developed by UCLA’s Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare and fatal immune disorder, with no serious complications ...
Scientists have made major progress in treating congenital deafness using gene therapy. This approach repairs hearing loss by fixing the root cause—a faulty gene known as OTOF. The OTOF gene creates ...
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Gene Therapy Successfully Revives Immune System In Children With Rare Disorder, Reveals Study
A groundbreaking study reveals that an experimental gene therapy has successfully restored immune system function in children with adenosine deaminase deficiency severe combined immunodeficiency ...
What Is Itvisma, and Why Does It Matter? Itvisma (onasemnogene abeparvovec-brve) is a gene therapy that the FDA has approved to treat spinal muscular atrophy (SMA) in adults, teens, and children 2 or ...
ORLANDO, Fla. — Exagamglogene autotemcel (exa-cel, Casgevy), a CRISPR/Cas9 gene-editing therapy, showed efficacy in children aged 5-11 years with transfusion-dependent beta-thalassemia (TDT) or sickle ...
Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...
On Monday, the U.S. Food and Drug Administration (FDA) approved Novartis AG’s (NYSE: NVS) Itvisma (onasemnogene abeparvovec-brve) for children two years and older, teens, and adults with spinal ...
A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated children, offering a lasting, donor-free solution to ADA-SCID, known as the ...
Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...
EAST HANOVER, N.J., Nov. 24, 2025 /PRNewswire/ -- Novartis today announced that the US Food and Drug Administration (FDA) has approved Itvisma® (onasemnogene abeparvovec-brve) for the treatment of ...
CLEVELAND, Ohio and AURORA, Colo., Oct. 08, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (ABEO) and Children's Hospital Colorado today announced activation of Children's Colorado as the newest ...
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