Browsing Tag
Gene Therapy
13 posts
How gene therapy is changing Logan’s life
[ad_1] TAMPA, Fla. (WGEM) – It’s a devastating diagnosis that changes a child’s life and their family’s, forever.…
May 14, 2025
Inhalable Gene Therapies? Here’s How They Could Work
[ad_1] We’ve come a long way in treatments for cystic fibrosis (CF) that extend lifespans. In 2008, the…
May 1, 2025
New genetic medicine offers life-changing vision improvements for blind children
[ad_1] Four young children have gained life-changing improvements in sight following treatment with a pioneering new genetic medicine…
February 22, 2025
StitchR technology delivers large genes for muscular dystrophy treatment
[ad_1] Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is…
November 15, 2024
In Gene Therapy for RP, LCA, the Earlier, the Better
[ad_1] The only approved ocular gene therapy, which targets a specific gene mutation in two forms of inherited…
October 4, 2024
Arun Upadhyay, PhD, on Results from IRD Gene Therapy OCU400’s Phase 1/2 Trial
[ad_1] “I would like to highlight that currently we are enrolling patients in our OCU400 phase 3 study……
October 1, 2024
Scientists explore early life factors contributing to SMA
[ad_1] Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although…
July 27, 2024
Gene therapy milestone at SickKids offers hope for children with SPG50 neurodegenerative disorder
[ad_1] When Michael Pirovolakis received an individualized gene therapy in a single-patient clinical trial at The Hospital for…
June 29, 2024
New curative treatment for sickle cell disease shows promising results in international trial
[ad_1] Vanderbilt University Medical Center (VUMC) researchers are touting data from a multicenter, international phase 2 clinical trial…
June 26, 2024
New gene editing approach could revolutionize the treatment of blood disorders
[ad_1] In a step forward in the development of genetic medicines, researchers at Children’s Hospital of Philadelphia (CHOP)…
July 28, 2023