Therapeutics in paediatric genetic diseases: current and future landscape

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...

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