FDA approves first gene therapy for deafness
#GeneTherapy #DeafnessThe FDA approved the first gene therapy for deafness, restoring hearing in people born with a rare OTOF gene defect. Treatment uses viruses to deliver a healthy gene, with 80% of patients gaining significant hearing. It's hailed as a landmark for hearing loss treatment. read more
Gene Therapy Restores Hearing in Deaf Children
#GeneTherapy #DeafnessTreatmentAn experimental gene therapy safely restored hearing to people born with a rare genetic deafness. The largest study to date shows lasting, near-normal results for many patients, offering a transformative new treatment option. read more
#GeneTherapy #CanavanDisease
Ciro Lovato saw improvement at Dayton Children’s hospital after moving from Guatemala to Ohio for gene therapy trial due to Canavan disease. Gene therapy trial involves virus delivering DNA into brain cells. Parents noted positive changes in Ciro. Funding challenges and selective trial eligibility remain. read more