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Recommendations for the design of small population clinical trials.
Day S, Jonker AH, Lau LPL, Hilgers RD, Irony I, Larsson K, Roes KC, Stallard N. Day S, et al. Among authors: larsson k. Orphanet J Rare Dis. 2018 Nov 6;13(1):195. doi: 10.1186/s13023-018-0931-2. Orphanet J Rare Dis. 2018. PMID: 30400970 Free PMC article.
Are products with an orphan designation for oncology indications different from products for other rare indications? A retrospective analysis of European orphan designations granted between 2002-2012.
Pauwels K, Huys I, Casteels M, Larsson K, Voltz C, Penttila K, Morel T, Simoens S. Pauwels K, et al. Among authors: larsson k. Orphanet J Rare Dis. 2017 Feb 16;12(1):36. doi: 10.1186/s13023-017-0578-4. Orphanet J Rare Dis. 2017. PMID: 28209180 Free PMC article.
Nonclinical data supporting orphan medicinal product designations in the area of rare infectious diseases.
Sheean ME, Malikova E, Duarte D, Capovilla G, Fregonese L, Hofer MP, Magrelli A, Mariz S, Mendez-Hermida F, Nistico R, Leest T, Sipsas NV, Tsigkos S, Vitezic D, Larsson K, Sepodes B, Stoyanova-Beninska V. Sheean ME, et al. Among authors: larsson k. Drug Discov Today. 2020 Feb;25(2):274-291. doi: 10.1016/j.drudis.2019.10.015. Epub 2019 Nov 5. Drug Discov Today. 2020. PMID: 31704277 Free article. Review.
Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.
Tambuyzer E, Vandendriessche B, Austin CP, Brooks PJ, Larsson K, Miller Needleman KI, Valentine J, Davies K, Groft SC, Preti R, Oprea TI, Prunotto M. Tambuyzer E, et al. Among authors: larsson k. 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.
Nonclinical data supporting orphan medicinal product designations: lessons from rare neurological conditions.
Sheean ME, Stoyanova-Beninska V, Capovilla G, Duarte D, Hofer MP, Hoffmann M, Magrelli A, Mariz S, Tsigkos S, Shaili E, Polsinelli B, Ricciardi M, Bonelli M, Balabanov P, Larsson K, Sepodes B. Sheean ME, et al. Among authors: larsson k. Drug Discov Today. 2018 Jan;23(1):26-48. doi: 10.1016/j.drudis.2017.09.015. Epub 2017 Oct 4. Drug Discov Today. 2018. PMID: 28987289 Review.
Regulatory Standards in Orphan Medicinal Product Designation in the EU.
Tsigkos S, Mariz S, Sheean ME, Larsson K, Magrelli A, Stoyanova-Beninska V. Tsigkos S, et al. Among authors: larsson k. Front Med (Lausanne). 2021 Jun 25;8:698534. doi: 10.3389/fmed.2021.698534. eCollection 2021. Front Med (Lausanne). 2021. PMID: 34249982 Free PMC article. Review.
Assessment of significant benefit for orphan medicinal products by European regulators may support subsequent relative effectiveness assessments by health technology assessment organizations.
Vreman RA, de Ruijter AS, Zawada A, Tafuri G, Stoyanova-Beninska V, O'Connor D, Naumann-Winter F, Wolter F, Mantel-Teeuwisse AK, Leufkens HGM, Sidiropoulos I, Larsson K, Goettsch WG. Vreman RA, et al. Among authors: larsson k. Drug Discov Today. 2020 Jul;25(7):1223-1231. doi: 10.1016/j.drudis.2020.04.012. Epub 2020 Apr 25. Drug Discov Today. 2020. PMID: 32344040 Free article. Review.
1,065 results