Therapeutics in paediatric genetic diseases: current and future landscape

• Main Authors: Koh, Ai Ling, Jamuar, Saumya Shekhar
• Other Authors: Lee Kong Chian School of Medicine (LKCMedicine)
• Format: Article
• Language: English
• Published: 2023
• Subjects: Science::Medicine; Antisense Oligonucleotide; Gene Therapy
• Online Access: https://hdl.handle.net/10356/169411
• Institution: Nanyang Technological University

There are more than 7,000 paediatric genetic diseases (PGDs) but less than 5% have treatment options. Treatment strategies targeting different levels of the biological process of the disease have led to optimal health outcomes in a subset of patients with PGDs, where treatment is available. In the p...