Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My Custom Filters

Publication date

Text availability

Article attribute

Article type

Additional filters

Article Language

Species

Sex

Age

Other

Search Results

1,065 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Publication Date timeline is not available.
Page 1
Defining orphan conditions in the context of the European orphan regulation: challenges and evolution.
O'Connor DJ, Sheean ME, Hofer MP, Tsigkos S, Mariz S, Fregonese L, Larsson K, Hivert V, Westermark K, Naumann-Winter F, Stoyanova-Beninska V, Barišić I, Capovilla G, Magrelli A, Sepodes B. O'Connor DJ, et al. Among authors: larsson k. Nat Rev Drug Discov. 2019 Jul;18(7):479-480. doi: 10.1038/nrd.2018.128. Nat Rev Drug Discov. 2019. PMID: 30940922 No abstract available.
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.
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.
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.
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.
Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit-The EU Regulator's Perspective.
Sheean ME, Naumann-Winter F, Capovilla G, Kalland ME, Malikova E, Mariz S, Matusevicius D, Nistico R, Schwarzer-Daum B, Tsigkos S, Tzogani K, Larsson K, Magrelli A, Stoyanova-Beninska V. Sheean ME, et al. Among authors: larsson k. Front Med (Lausanne). 2021 Aug 27;8:744625. doi: 10.3389/fmed.2021.744625. eCollection 2021. Front Med (Lausanne). 2021. PMID: 34513895 Free PMC article.
1,065 results