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Page 1
Correction to: How is the Pharmaceutical Industry Structured to Optimize Pediatric Drug Development? Existing Pediatric Structure Models and Proposed Recommendations for Structural Enhancement.
Severin T, Corriol-Rohou S, Bucci-Rechtweg C, An Haack K, Fuerst-Recktenwald S, Lepola P, Norjavaara E, Dehlinger-Kremer M; EFGCP Children’s Medicines Working Party; Haertter S, Cheung SYA; IQ Pediatric Working Group. Severin T, et al. Among authors: an haack k. Ther Innov Regul Sci. 2020 Sep;54(5):1085. doi: 10.1007/s43441-020-00152-0. Ther Innov Regul Sci. 2020. PMID: 32865808 Free PMC article.
Measurement Properties of 2 Novel PROs, the Pompe Disease Symptom Scale and Pompe Disease Impact Scale, in the COMET Study.
Dimachkie MM, Kishnani PS, Ivanescu C, Flore G, Gwaltney C, van der Beek NAME, Hamed A, An Haack K, Pollissard L, Baranowski E, Sparks SE, DasMahapatra P; for COMET Study Group. Dimachkie MM, et al. Among authors: an haack k. Neurol Clin Pract. 2023 Oct;13(5):e200181. doi: 10.1212/CPJ.0000000000200181. Epub 2023 Aug 8. Neurol Clin Pract. 2023. PMID: 37559825 Free PMC article.
How is the Pharmaceutical Industry Structured to Optimize Pediatric Drug Development? Existing Pediatric Structure Models and Proposed Recommendations for Structural Enhancement.
Severin T, Corriol-Rohou S, Bucci-Rechtweg C, An Haack K, Fuerst-Recktenwald S, Lepola P, Norjavaara E, Dehlinger-Kremer M; EFGCP Children’s Medicines Working Party; Haertter S, Cheung SYA; IQ Pediatric Working Group. Severin T, et al. Among authors: an haack k. Ther Innov Regul Sci. 2020 Sep;54(5):1076-1084. doi: 10.1007/s43441-020-00116-4. Epub 2020 Feb 6. Ther Innov Regul Sci. 2020. PMID: 32030690 Free PMC article.
Development and content validation of the Pediatric Oral Medicines Acceptability Questionnaires (P-OMAQ): patient-reported and caregiver-reported outcome measures.
Turner-Bowker DM, An Haack K, Krohe M, Yaworsky A, Vivas N, Kelly M, Chatterjee G, Chaston E, Mann E, Reaney M. Turner-Bowker DM, et al. Among authors: an haack k. J Patient Rep Outcomes. 2020 Oct 1;4(1):80. doi: 10.1186/s41687-020-00246-1. J Patient Rep Outcomes. 2020. PMID: 33000327 Free PMC article.
Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study.
Hahn SH, Kronn D, Leslie ND, Pena LDM, Tanpaiboon P, Gambello MJ, Gibson JB, Hillman R, Stockton DW, Day JW, Wang RY, An Haack K, Shafi R, Sparks S, Zhao Y, Wilson C, Kishnani PS; Pompe ADVANCE Study Consortium. Hahn SH, et al. Among authors: an haack k. Genet Med. 2018 Oct;20(10):1284-1294. doi: 10.1038/gim.2018.2. Epub 2018 Mar 22. Genet Med. 2018. PMID: 29565424 Free article. Clinical Trial.
Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease.
Kishnani PS, Gibson JB, Gambello MJ, Hillman R, Stockton DW, Kronn D, Leslie ND, Pena LDM, Tanpaiboon P, Day JW, Wang RY, Goldstein JL, An Haack K, Sparks SE, Zhao Y, Hahn SH; Pompe ADVANCE Study Consortium. Kishnani PS, et al. Among authors: an haack k. Genet Med. 2019 Nov;21(11):2543-2551. doi: 10.1038/s41436-019-0527-9. Epub 2019 May 14. Genet Med. 2019. PMID: 31086307 Free PMC article.
Cardiac responses in paediatric Pompe disease in the ADVANCE patient cohort.
Byrne BJ, Colan SD, Kishnani PS, Foster MC, Sparks SE, Gibson JB, An Haack K, Stockton DW, Peña LDM, Hahn SH, Johnson J, Tanpaiboon PX, Leslie ND, Kronn D, Hillman RE, Wang RY; Pompe ADVANCE Study Consortium. Byrne BJ, et al. Among authors: an haack k. Cardiol Young. 2022 Mar;32(3):364-373. doi: 10.1017/S1047951121002079. Epub 2021 Aug 23. Cardiol Young. 2022. PMID: 34420548 Clinical Trial.
23 results