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: Ai Ling Koh, Saumya Shekhar Jamuar
Format: Article
Language:English
Published: Wolters Kluwer – Medknow Publications 2023-01-01
Series:Singapore Medical Journal
Subjects:
Online Access:https://journals.lww.com/10.4103/singaporemedj.SMJ-2021-376
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