Hosted on MSN

Gene Therapy Shows Lasting Benefit for Children With Rare Disorder

For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational gene therapy showed sustained clinical efficacy and safety at a median ...
News Medical

Gene therapy shows long-term success in children with rare immune disorder

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 ...
EurekAlert!

Landmark gene therapy study shows safety for children

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 ...
University of California

Gene therapy delivers lasting immune protection in children with rare disorder

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 ...
News-Medical.Net on MSN

Advances in hemophilia gene therapy bring hope for pediatric patients

In the past three years, gene therapy has reshaped what's possible in hemophilia treatment for patients 18 and older. But a ...
KPLC

Gene therapy offering hope to kids with genetic disorders

TAMPA, Fla. (Ivanhoe Newswire) - It’s a devastating diagnosis that changes a child’s life, and their family’s, forever. Duchenne Muscular Dystrophy, or DMD, is a rare and fatal genetic disease that ...
Medical Xpress

Gene therapy improves movement in kids with spinal muscular atrophy

A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...
WebMD

Itvisma: FDA Approves One-Time Gene Therapy for Spinal Muscular Atrophy in Children 2 or Older, Teens, and Adults

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 ...

Baby who received personalized gene editing therapy at CHOP takes first steps

A baby born with a rare genetic disorder has taken his first steps after receiving a groundbreaking, personalized gene ...

My son was given world's most expensive gene therapy drug - now he can walk

Edward, from Colchester, has spinal muscular atrophy (SMA) which means he lacks a protein vital for muscle development. He ...
Morningstar

Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)

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 ...
Medical Xpress

Longevity gene from supercentenarians offers hope for disease that causes rapid aging in children

A new breakthrough in a rare genetic disease which causes children to age rapidly has been discovered using 'longevity genes' found in people who live exceptionally long lives—over 100 years old. The ...