Achieving big with small: quantitative clinical pharmacology tools for drug development in pediatric rare diseases
Author:
Publisher
Springer Science and Business Media LLC
Subject
Pharmacology
Link
https://link.springer.com/content/pdf/10.1007/s10928-023-09863-x.pdf
Reference131 articles.
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3. Smits RM, Vissers E, Te Pas R, Roebbers N, Feitz WF, van Rooij IA, de Blaauw I, Verhaak CM (2022) Common needs in uncommon conditions: a qualitative study to explore the need for care in pediatric patients with rare diseases. Orphanet J Rare Dis 17(1):1–9
4. Lalonde E, Rentas S, Lin F, Dulik MC, Skraban CM, Spinner NB (2020) Genomic diagnosis for pediatric disorders: revolution and evolution. Front Pediatr 8:373
5. Hartley T, Lemire G, Kernohan KD, Howley HE, Adams DR, Boycott KM (2020) New diagnostic approaches for undiagnosed rare genetic diseases. Annu Rev Genom Hum Genet 21(1):351–372
Cited by 2 articles. 订阅此论文施引文献 订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献
1. Getting the Dose Right in Drug Development for Rare Diseases: Barriers and Enablers;Clinical Pharmacology & Therapeutics;2024-08-16
2. Integrated Applied Clinical Pharmacology in the Advancement of Rare and Ultra‐Rare Disease Therapeutics;Clinical Pharmacology & Therapeutics;2024-07-22
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