An investigational gene therapy showed sustained clinical efficacy for children born with ADA-SCID. In a study of 62 children, overall survival after treatment was 100% and event-free survival was 95% ...

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 ...

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 ...

A groundbreaking study reveals that an experimental gene therapy has successfully restored immune system function in children with adenosine deaminase deficiency severe combined immunodeficiency ...

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 ...

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 ...

This time, her baby did test positive for SMA type 1, a severe neurodegenerative genetic condition that typically results in ...

Thanks to advances in imaging and diagnostic technologies, clinicians can now detect many genetic disorders in the womb, ...

Groundbreaking Phase 1/2a clinical trials co-led by Linda Laux, MD , from Ann & Robert H. Lurie Children's Hospital of Chicago, show that the first ...

July 21 (Reuters) - Children's Hospital Los Angeles, citing recent U.S. Food and Drug Administration actions, on Monday said it has paused usage of Sarepta Therapeutics' (SRPT.O), opens new tab gene ...