Tag: gene therapy delivery method safety for pediatric rare disease

2024 Expert Guide to Rare Disease Gene Therapy: In Vivo vs Ex Vivo Differences, Pediatric Patient Safety, Local Ocular & Systemic Metabolic Delivery Benefits & Latest Advancements

Per 2024 FDA, 2023 NIH, and 2024 National Organization for Rare Disorders (NORD) data, 3 new rare disease gene therapies earned FDA approval this year, with 75% of eligible therapies qualifying for fast-track access for pediatric patients. This November 2024 updated expert buying guide breaks down in vivo vs ex vivo gene therapy differences, safety