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Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.
Nat Rev Drug Discov. 2020 Feb;19(2):93-111. doi: 10.1038/s41573-019-0049-9. Epub 2019 Dec 13.
Nat Rev Drug Discov. 2020.
PMID: 31836861
Review.
FDA's Office of Orphan Products Development: providing incentives to promote the development of products for rare diseases.
Patel S, Miller Needleman KI.
Patel S, et al. Among authors: miller needleman ki.
J Pharmacokinet Pharmacodyn. 2019 Oct;46(5):387-393. doi: 10.1007/s10928-019-09645-4. Epub 2019 Jul 5.
J Pharmacokinet Pharmacodyn. 2019.
PMID: 31278433
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Precision Medicines' Impact on Orphan Drug Designation.
Mueller CM, Rao GR, Miller Needleman KI.
Mueller CM, et al. Among authors: miller needleman ki.
Clin Transl Sci. 2019 Nov;12(6):633-640. doi: 10.1111/cts.12667. Epub 2019 Jul 26.
Clin Transl Sci. 2019.
PMID: 31297924
Free PMC article.
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FDA orphan products clinical trial grants: assessment of outcomes and impact on rare disease product development.
Miller KL, Mueller C, Liu G, Miller Needleman KI, Maynard J.
Miller KL, et al. Among authors: miller needleman ki.
Orphanet J Rare Dis. 2020 Sep 3;15(1):234. doi: 10.1186/s13023-020-01514-5.
Orphanet J Rare Dis. 2020.
PMID: 32883327
Free PMC article.
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The effect of the COVID-19 pandemic on US Food and Drug Administration-funded clinical trials and natural history studies for rare diseases.
Miller KL, Miller Needleman KI.
Miller KL, et al. Among authors: miller needleman ki.
Contemp Clin Trials. 2023 Jun;129:107198. doi: 10.1016/j.cct.2023.107198. Epub 2023 Apr 15.
Contemp Clin Trials. 2023.
PMID: 37068589
Free PMC article.
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