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 NCBI Filters

Text availability

Article attribute

Article type

Publication date

Search Results

36 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
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: magrelli a. Eur J Ophthalmol. 2024 Mar 18:11206721241236214. doi: 10.1177/11206721241236214. Online ahead of print. Eur J Ophthalmol. 2024. PMID: 38500388 Free 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: magrelli a. 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: magrelli a. 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.
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: magrelli a. 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: magrelli a. 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.
The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products.
Palomo GM, Pose-Boirazian T, Naumann-Winter F, Costa E, Duarte DM, Kalland ME, Malikova E, Matusevicius D, Vitezic D, Larsson K, Magrelli A, Stoyanova-Beninska V, Mariz S. Palomo GM, et al. Among authors: magrelli a. Mol Ther. 2023 Dec 6;31(12):3414-3423. doi: 10.1016/j.ymthe.2023.09.020. Epub 2023 Oct 4. Mol Ther. 2023. PMID: 37794679 Free article.
Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation.
Kalland ME, Pose-Boirazian T, Palomo GM, Naumann-Winter F, Costa E, Matusevicius D, Duarte DM, Malikova E, Vitezic D, Larsson K, Magrelli A, Stoyanova-Beninska V, Mariz S. Kalland ME, et al. Among authors: magrelli a. Gene Ther. 2024 Mar 14. doi: 10.1038/s41434-024-00446-0. Online ahead of print. Gene Ther. 2024. PMID: 38480914
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: magrelli a. 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.
36 results