UMass Chan licenses gene therapy for rare genetic disorder to Raiden Science Foundation
Raiden Pham, who was diagnosed with UBA5 disorder in 2021, with his mother Linda Pham. Photo courtesy of Tommy and Linda...
disorder
New hope for children with devastating rare genetic disorder, thanks to world-first research in Manchester
The parents of a three-year-old boy born with a devastating, life-limiting genetic condition say they are now excited for his...
Experimental gene therapy restores immune system function in children with genetic immune disorder
An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has restored and maintained...
Depressive disorder (depression)
Key facts Depression is a common mental disorder.Globally, an estimated 5.7% of adults suffer from depression.More women are affected by...
Gene therapy trial offers breakthrough for rare genetic disorder
Credit: Pixabay/CC0 Public Domain A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising...
A bespoke gene-editing treatment for a baby born with a life-threatening disorder : Shots
KJ Muldoon, who was born at Children's Hospital of Philadelphia in August, reaches out to doctors after being treated for...
ASH: Gene therapy for rare genetic disorder, molecular insights into leukemia growth and more
Researchers from the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA will present their...
Gene therapy hailed as ‘medical magic wand’ for hereditary swelling disorder | Medical research
A groundbreaking gene therapy has been hailed as a “medical magic wand” after the treatment transformed the lives of patients...
Gene therapy milestone at SickKids offers hope for children with SPG50 neurodegenerative disorder
When Michael Pirovolakis received an individualized gene therapy in a single-patient clinical trial at The Hospital for Sick Children (SickKids)...
