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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
2004 1
2005 2
2006 4
2007 1
2008 5
2009 3
2010 2
2011 2
2014 1
2017 5
2018 4
2019 5
2020 4
2021 3
2022 3
2023 3
2024 2

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41 results

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Page 1
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.
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.
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.
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.
A Randomized Controlled Clinical Trial of Transanal Irrigation Versus Conservative Treatment in Patients With Low Anterior Resection Syndrome After Rectal Cancer Surgery.
Pieniowski EHA, Bergström CM, Nordenvall CAM, Westberg KS, Johar AM, Tumlin Ekelund SF, Larsson KR, Pekkari KJ, Jansson Palmer GC, Lagergren P, Abraham-Nordling M. Pieniowski EHA, et al. Among authors: larsson kr. Ann Surg. 2023 Jan 1;277(1):30-37. doi: 10.1097/SLA.0000000000005482. Epub 2022 Jul 7. Ann Surg. 2023. PMID: 35797618 Clinical Trial.
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.
Inherited retinal dystrophies and orphan designations in the European Union.
Moseley J, Leest T, Larsson K, Magrelli A, Stoyanova-Beninska V. Moseley J, et al. Among authors: larsson k. Eur J Ophthalmol. 2024 Mar 18:11206721241236214. doi: 10.1177/11206721241236214. Online ahead of print. Eur J Ophthalmol. 2024. PMID: 38500388 Free article. Review.
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.
41 results