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Towards the international interoperability of clinical research networks for rare diseases: recommendations from the IRDiRC Task Force.
Nabbout R, Zanello G, Baker D, Black L, Brambilla I, Buske OJ, Conklin LS, Davies EH, Julkowska D, Kim Y, Klopstock T, Nakamura H, Nielsen KG, Pariser AR, Pastor JC, Scarpa M, Smith M, Taruscio D, Groft S. Nabbout R, et al. Among authors: conklin ls. Orphanet J Rare Dis. 2023 May 9;18(1):109. doi: 10.1186/s13023-023-02650-4. Orphanet J Rare Dis. 2023. PMID: 37161573 Free PMC article.
Innovations in Pediatric Therapeutics Development: Principles for the Use of Bridging Biomarkers in Pediatric Extrapolation.
Fleming TR, Garnett CE, Conklin LS, Corriol-Rohou S, Hariharan S, Hsu D, Mueller-Velten G, Mulugeta Y, Portman R, Rothmann MD, Stockbridge NL, Wandel S, Zhang J, Yao L. Fleming TR, et al. Among authors: conklin ls. Ther Innov Regul Sci. 2023 Jan;57(1):109-120. doi: 10.1007/s43441-022-00445-6. Epub 2022 Sep 3. Ther Innov Regul Sci. 2023. PMID: 36057747 Free PMC article.
The Role of Master Protocols in Pediatric Drug Development.
Nelson RM, Conklin LS, Komocsar WJ, Chen F, Williamson F, Crandall WV. Nelson RM, et al. Among authors: conklin ls. Ther Innov Regul Sci. 2022 Nov;56(6):895-902. doi: 10.1007/s43441-022-00448-3. Epub 2022 Sep 1. Ther Innov Regul Sci. 2022. PMID: 36045315 Free PMC article. Review.
Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.
Smith EC, Conklin LS, Hoffman EP, Clemens PR, Mah JK, Finkel RS, Guglieri M, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, Kerchner L, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, Gordish-Dressman H, Hagerty L, Dang UJ, Damsker JM, Schwartz BD, Mengle-Gaw LJ, McDonald CM; CINRG VBP15 and DNHS Investigators. Smith EC, et al. Among authors: conklin ls. PLoS Med. 2020 Sep 21;17(9):e1003222. doi: 10.1371/journal.pmed.1003222. eCollection 2020 Sep. PLoS Med. 2020. PMID: 32956407 Free PMC article. Clinical Trial.
41 results