Leufkens HGM - Search Results

1

Successes and pitfalls in orphan drug development for sickle cell disease.

Costa E, Isgrò A, de Montalembert M, Leufkens HGM, Ware RE, De Franceschi L. Costa E, et al. Among authors: leufkens hgm. Blood Adv. 2024 May 28;8(10):2455-2465. doi: 10.1182/bloodadvances.2023011730. Blood Adv. 2024. PMID: 38522095 Free PMC article.

2

The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis.

Costa E, Girotti S, Pauro F, Leufkens HGM, Cipolli M. Costa E, et al. Among authors: leufkens hgm. Orphanet J Rare Dis. 2022 May 7;17(1):188. doi: 10.1186/s13023-022-02350-5. Orphanet J Rare Dis. 2022. PMID: 35525974 Free PMC article.

3

Emergent treatments for β-thalassemia and orphan drug legislations.

Costa E, Cappellini MD, Rivella S, Chilin A, Alessi E, Riccaboni M, Leufkens HGM, Luzzatto L. Costa E, et al. Among authors: leufkens hgm. Drug Discov Today. 2022 Nov;27(11):103342. doi: 10.1016/j.drudis.2022.103342. Epub 2022 Sep 1. Drug Discov Today. 2022. PMID: 36058507 Free article. Review.

4

Traits, trends and hits of orphan drug designations in cystic fibrosis.

Costa E, Girotti S, van den Ham HA, Cipolli M, van der Ent CK, Taylor-Cousar JL, Leufkens HGM. Costa E, et al. Among authors: leufkens hgm. J Cyst Fibros. 2023 Sep;22(5):949-957. doi: 10.1016/j.jcf.2023.07.006. Epub 2023 Jul 27. J Cyst Fibros. 2023. PMID: 37507282 Free article.

5

Globalization in clinical drug development for sickle cell disease.

Costa E, Ware RE, Tshilolo L, Makani J, Leufkens HGM, Luzzatto L. Costa E, et al. Among authors: leufkens hgm. Am J Hematol. 2025 Jan;100(1):4-9. doi: 10.1002/ajh.27525. Epub 2024 Nov 12. Am J Hematol. 2025. PMID: 39530361 Free PMC article. No abstract available.

6

Translation of rare disease research into orphan drug development: disease matters.

Heemstra HE, van Weely S, Büller HA, Leufkens HG, de Vrueh RL. Heemstra HE, et al. Drug Discov Today. 2009 Dec;14(23-24):1166-73. doi: 10.1016/j.drudis.2009.09.008. Epub 2009 Oct 7. Drug Discov Today. 2009. PMID: 19818412 Review.

7

Orphan drug development across Europe: bottlenecks and opportunities.

Heemstra HE, de Vrueh RL, van Weely S, Büller HA, Leufkens HG. Heemstra HE, et al. Drug Discov Today. 2008 Aug;13(15-16):670-6. doi: 10.1016/j.drudis.2008.05.001. Epub 2008 Jun 24. Drug Discov Today. 2008. PMID: 18583178 Review.

8

Evaluation of oncology drugs at the European Medicines Agency and US Food and Drug Administration: when differences have an impact on clinical practice.

Trotta F, Leufkens HG, Schellens JH, Laing R, Tafuri G. Trotta F, et al. J Clin Oncol. 2011 Jun 1;29(16):2266-72. doi: 10.1200/JCO.2010.34.1248. Epub 2011 May 2. J Clin Oncol. 2011. PMID: 21537038

9

Determinants for successful marketing authorisation of orphan medicinal products in the EU.

Putzeist M, Heemstra HE, Garcia JL, Mantel-Teeuwisse AK, Gispen-De Wied CC, Hoes AW, Leufkens HG. Putzeist M, et al. Drug Discov Today. 2012 Apr;17(7-8):352-8. doi: 10.1016/j.drudis.2011.10.027. Epub 2011 Nov 7. Drug Discov Today. 2012. PMID: 22094244 Review.

10

EU marketing authorization review of orphan and non-orphan drugs does not differ.

Putzeist M, Mantel-Teeuwisse AK, Llinares J, Gispen-De Wied CC, Hoes AW, Leufkens HG. Putzeist M, et al. Drug Discov Today. 2013 Oct;18(19-20):1001-6. doi: 10.1016/j.drudis.2013.06.012. Epub 2013 Jul 5. Drug Discov Today. 2013. PMID: 23835230 Review.

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