Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...
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 ...
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 gene therapy study published recently in the journal Nature Medicine showed promising results in restoring hearing in patients of all ages with genetic hearing loss and congenital deafness. The ...
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 ...
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Experimental gene therapy successfully treats dozens of children with rare fatal disorder
An experimental gene therapy has successfully treated dozens of children with a rare and fatal immune disorder, a promising sign that it could one day be defeated. The study included 62 children with ...
Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...
Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts treated with cochlear implantation in a cohort study from China. Gene ...
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 ...
A single dose of a new formulation of the gene replacement therapy intrathecal onasemnogene abeparvovec ( OAV101 IT) improved movement ability in teens and children older than 2 years with spinal ...
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