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Showing 1 to 12 of 31 entries
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Product development partnerships (PDPs) for neglected diseases: considerations on governance.

Health economics, policy, and law

Sorenson C.
PMID: 19099614
Health Econ Policy Law. 2009 Jan;4:1-10. doi: 10.1017/S1744133108004702.

No abstract available.

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Sorenson C. Product development partnerships (PDPs) for neglected diseases: considerations on governance. Health Econ Policy Law. 2009;4:1-10doi: 10.1017/S1744133108004702.
Sorenson, C. (2009). Product development partnerships (PDPs) for neglected diseases: considerations on governance. Health economics, policy, and law, 41-10. https://doi.org/10.1017/S1744133108004702
Sorenson, Corinna. "Product development partnerships (PDPs) for neglected diseases: considerations on governance." Health economics, policy, and law vol. 4 (2009): 1-10. doi: https://doi.org/10.1017/S1744133108004702
Sorenson C. Product development partnerships (PDPs) for neglected diseases: considerations on governance. Health Econ Policy Law. 2009 Jan;4:1-10. doi: 10.1017/S1744133108004702. PMID: 19099614.
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[Development of new indications for old products: difficulties and search for solutions].

Presse medicale (Paris, France : 1983)

Duguet C.
PMID: 22483765
Presse Med. 2012 May;41:S34-6. doi: 10.1016/j.lpm.2012.02.012. Epub 2012 Apr 06.

No abstract available.

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Duguet C. Développement d'indications nouvelles pour des molécules anciennes : difficultés et recherche de solutions. [Development of new indications for old products: difficulties and search for solutions]. Presse Med. 2012;41:S34-6doi: 10.1016/j.lpm.2012.02.012.
Duguet, C. (2012). Développement d'indications nouvelles pour des molécules anciennes : difficultés et recherche de solutions. [Development of new indications for old products: difficulties and search for solutions]. Presse medicale (Paris, France : 1983), 41S34-6. https://doi.org/10.1016/j.lpm.2012.02.012
Duguet, Christophe. "Développement d'indications nouvelles pour des molécules anciennes : difficultés et recherche de solutions." [Development of new indications for old products: difficulties and search for solutions]. Presse medicale (Paris, France : 1983) vol. 41 (2012): S34-6. doi: https://doi.org/10.1016/j.lpm.2012.02.012
Duguet C. Développement d'indications nouvelles pour des molécules anciennes : difficultés et recherche de solutions. [Development of new indications for old products: difficulties and search for solutions]. Presse Med. 2012 May;41:S34-6. doi: 10.1016/j.lpm.2012.02.012. Epub 2012 Apr 06. French. PMID: 22483765.
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A case study of an adaptive design for a clinical trial with 2 doses and 2 endpoints in a rare disease area.

Pharmaceutical statistics

Quan H, Xu Y, Chen Y, Gao L, Chen X.
PMID: 30221446
Pharm Stat. 2018 Nov;17(6):797-810. doi: 10.1002/pst.1902. Epub 2018 Sep 16.

Patient recruitment is challenging in rare disease clinical trials. To save time and resources, an inferential seamless phase II/III clinical trial design is considered for a clinical trial in a rare disease area. In particular, 2 doses compared to...

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Quan H, Xu Y, Chen Y, et al. A case study of an adaptive design for a clinical trial with 2 doses and 2 endpoints in a rare disease area. Pharm Stat. 2018;17(6):797-810doi: 10.1002/pst.1902.
Quan, H., Xu, Y., Chen, Y., Gao, L., & Chen, X. (2018). A case study of an adaptive design for a clinical trial with 2 doses and 2 endpoints in a rare disease area. Pharmaceutical statistics, 17(6), 797-810. https://doi.org/10.1002/pst.1902
Quan, Hui, et al. "A case study of an adaptive design for a clinical trial with 2 doses and 2 endpoints in a rare disease area." Pharmaceutical statistics vol. 17,6 (2018): 797-810. doi: https://doi.org/10.1002/pst.1902
Quan H, Xu Y, Chen Y, Gao L, Chen X. A case study of an adaptive design for a clinical trial with 2 doses and 2 endpoints in a rare disease area. Pharm Stat. 2018 Nov;17(6):797-810. doi: 10.1002/pst.1902. Epub 2018 Sep 16. PMID: 30221446.
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Duchenne muscular dystrophy: clinical trials and emerging tribulations.

Current opinion in neurology

Shieh PB.
PMID: 26280938
Curr Opin Neurol. 2015 Oct;28(5):542-6. doi: 10.1097/WCO.0000000000000243.

PURPOSE OF REVIEW: This article reviewed the most recent clinical trials investigating potential treatments for Duchenne muscular dystrophy (DMD). In the development of these studies, investigators have encountered unexpected challenges that reveal limitations in our understanding of the clinical...

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Shieh PB. Duchenne muscular dystrophy: clinical trials and emerging tribulations. Curr Opin Neurol. 2015;28(5):542-6doi: 10.1097/WCO.0000000000000243.
Shieh, P. B. (2015). Duchenne muscular dystrophy: clinical trials and emerging tribulations. Current opinion in neurology, 28(5), 542-6. https://doi.org/10.1097/WCO.0000000000000243
Shieh, Perry B. "Duchenne muscular dystrophy: clinical trials and emerging tribulations." Current opinion in neurology vol. 28,5 (2015): 542-6. doi: https://doi.org/10.1097/WCO.0000000000000243
Shieh PB. Duchenne muscular dystrophy: clinical trials and emerging tribulations. Curr Opin Neurol. 2015 Oct;28(5):542-6. doi: 10.1097/WCO.0000000000000243. PMID: 26280938.
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Artificial Intelligence (AI) in Rare Diseases: Is the Future Brighter?.

Genes

Brasil S, Pascoal C, Francisco R, Dos Reis Ferreira V, Videira PA, Valadão AG.
PMID: 31783696
Genes (Basel). 2019 Nov 27;10(12). doi: 10.3390/genes10120978.

The amount of data collected and managed in (bio)medicine is ever-increasing. Thus, there is a need to rapidly and efficiently collect, analyze, and characterize all this information. Artificial intelligence (AI), with an emphasis on deep learning, holds great promise...

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Brasil S, Pascoal C, Francisco R, et al. Artificial Intelligence (AI) in Rare Diseases: Is the Future Brighter?. Genes (Basel). 2019;10(12)doi: 10.3390/genes10120978.
Brasil, S., Pascoal, C., Francisco, R., Dos Reis Ferreira, V., Videira, P. A., & Valadão, A. G. (2019). Artificial Intelligence (AI) in Rare Diseases: Is the Future Brighter?. Genes, 10(12), . https://doi.org/10.3390/genes10120978
Brasil, Sandra, et al. "Artificial Intelligence (AI) in Rare Diseases: Is the Future Brighter?." Genes vol. 10,12 (2019). doi: https://doi.org/10.3390/genes10120978
Brasil S, Pascoal C, Francisco R, Dos Reis Ferreira V, Videira PA, Valadão AG. Artificial Intelligence (AI) in Rare Diseases: Is the Future Brighter?. Genes (Basel). 2019 Nov 27;10(12). doi: 10.3390/genes10120978. PMID: 31783696; PMCID: PMC6947640.
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Rare diseases and omics-driven personalized medicine.

Croatian medical journal

Šimić G.
PMID: 31894912
Croat Med J. 2019 Dec 31;60(6):485-487.

No abstract available.

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Šimić G. Rare diseases and omics-driven personalized medicine. Croat Med J. 2019;60(6):485-487
Šimić, G. (2019). Rare diseases and omics-driven personalized medicine. Croatian medical journal, 60(6), 485-487.
Šimić, Goran. "Rare diseases and omics-driven personalized medicine." Croatian medical journal vol. 60,6 (2019): 485-487.
Šimić G. Rare diseases and omics-driven personalized medicine. Croat Med J. 2019 Dec 31;60(6):485-487. PMID: 31894912; PMCID: PMC6952900.
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The needs of the few.

Nature

[No authors listed]
PMID: 20613796
Nature. 2010 Jul 08;466(7303):160. doi: 10.1038/466160a.

No abstract available.

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The needs of the few. Nature. 2010;466(7303):160doi: 10.1038/466160a.
(2010). The needs of the few. Nature, 466(7303), 160. https://doi.org/10.1038/466160a
"The needs of the few." Nature vol. 466,7303 (2010): 160. doi: https://doi.org/10.1038/466160a
The needs of the few. Nature. 2010 Jul 08;466(7303):160. doi: 10.1038/466160a. PMID: 20613796.
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Problems with finding evidence for rare events.

Managed care interface

Kozma CM.
PMID: 15573803
Manag Care Interface. 2004 Nov;17(11):45-6.

No abstract available.

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Kozma CM. Problems with finding evidence for rare events. Manag Care Interface. 2004;17(11):45-6
Kozma, C. M. (2004). Problems with finding evidence for rare events. Managed care interface, 17(11), 45-6.
Kozma, Chris M. "Problems with finding evidence for rare events." Managed care interface vol. 17,11 (2004): 45-6.
Kozma CM. Problems with finding evidence for rare events. Manag Care Interface. 2004 Nov;17(11):45-6. PMID: 15573803.
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Characteristics of rare disease marketing applications associated with FDA product approvals 2006-2010.

Drug discovery today

Pariser AR, Slack DJ, Bauer LJ, Warner CA, Tracy LA.
PMID: 22564486
Drug Discov Today. 2012 Aug;17(15):898-904. doi: 10.1016/j.drudis.2012.04.011. Epub 2012 May 05.

New drug and biologic product marketing applications submitted to FDA's Center for Drug Evaluation and Research (CDER) between 2006 and 2010 were analyzed to identify rare disease application characteristics associated with higher approval rates. The results show that approval...

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Pariser AR, Slack DJ, Bauer LJ, et al. Characteristics of rare disease marketing applications associated with FDA product approvals 2006-2010. Drug Discov Today. 2012;17(15):898-904doi: 10.1016/j.drudis.2012.04.011.
Pariser, A. R., Slack, D. J., Bauer, L. J., Warner, C. A., & Tracy, L. A. (2012). Characteristics of rare disease marketing applications associated with FDA product approvals 2006-2010. Drug discovery today, 17(15), 898-904. https://doi.org/10.1016/j.drudis.2012.04.011
Pariser, Anne R, et al. "Characteristics of rare disease marketing applications associated with FDA product approvals 2006-2010." Drug discovery today vol. 17,15 (2012): 898-904. doi: https://doi.org/10.1016/j.drudis.2012.04.011
Pariser AR, Slack DJ, Bauer LJ, Warner CA, Tracy LA. Characteristics of rare disease marketing applications associated with FDA product approvals 2006-2010. Drug Discov Today. 2012 Aug;17(15):898-904. doi: 10.1016/j.drudis.2012.04.011. Epub 2012 May 05. PMID: 22564486.
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[Diagnostics and treatment of ANCA-associated vasculitis].

Ugeskrift for laeger

Keller KK, Nielsen BD, Hauge EM, Hansen IT.
PMID: 26471022
Ugeskr Laeger. 2015 Oct 05;177(41):V12140721.

The term anti-neutrophil cytoplasmic antibodies (ANCA)-associated vasculitis (AAV) covers a group of rare diseases with inflammation in the small vessels and associated with the presence of ANCA. The patients can present with signs and symptoms from almost all organs....

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Keller KK, Nielsen BD, Hauge EM, et al. Diagnostik og behandling afANCA-associeret vaskulitis. [Diagnostics and treatment of ANCA-associated vasculitis]. Ugeskr Laeger. 2015;177(41):V12140721
Keller, K. K., Nielsen, B. D., Hauge, E. M., & Hansen, I. T. (2015). Diagnostik og behandling afANCA-associeret vaskulitis. [Diagnostics and treatment of ANCA-associated vasculitis]. Ugeskrift for laeger, 177(41), V12140721.
Keller, Kresten Krarup, et al. "Diagnostik og behandling afANCA-associeret vaskulitis." [Diagnostics and treatment of ANCA-associated vasculitis]. Ugeskrift for laeger vol. 177,41 (2015): V12140721.
Keller KK, Nielsen BD, Hauge EM, Hansen IT. Diagnostik og behandling afANCA-associeret vaskulitis. [Diagnostics and treatment of ANCA-associated vasculitis]. Ugeskr Laeger. 2015 Oct 05;177(41):V12140721. Danish. PMID: 26471022.
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Challenges of rare diseases in China.

Lancet (London, England)

Dong D, Wang Y.
PMID: 27203652
Lancet. 2016 May 07;387(10031):1906. doi: 10.1016/S0140-6736(16)30418-4.

No abstract available.

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Dong D, Wang Y. Challenges of rare diseases in China. Lancet. 2016;387(10031):1906doi: 10.1016/S0140-6736(16)30418-4.
Dong, D., & Wang, Y. (2016). Challenges of rare diseases in China. Lancet (London, England), 387(10031), 1906. https://doi.org/10.1016/S0140-6736(16)30418-4
Dong, Dong, and Wang, Yiou. "Challenges of rare diseases in China." Lancet (London, England) vol. 387,10031 (2016): 1906. doi: https://doi.org/10.1016/S0140-6736(16)30418-4
Dong D, Wang Y. Challenges of rare diseases in China. Lancet. 2016 May 07;387(10031):1906. doi: 10.1016/S0140-6736(16)30418-4. PMID: 27203652.
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First CD123-targeted drug approved after wowing in rare cancer.

Nature biotechnology

Dolgin E.
PMID: 30833771
Nat Biotechnol. 2019 Mar;37(3):202-203. doi: 10.1038/s41587-019-0056-8.

No abstract available.

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Dolgin E. First CD123-targeted drug approved after wowing in rare cancer. Nat Biotechnol. 2019;37(3):202-203doi: 10.1038/s41587-019-0056-8.
Dolgin, E. (2019). First CD123-targeted drug approved after wowing in rare cancer. Nature biotechnology, 37(3), 202-203. https://doi.org/10.1038/s41587-019-0056-8
Dolgin, Elie. "First CD123-targeted drug approved after wowing in rare cancer." Nature biotechnology vol. 37,3 (2019): 202-203. doi: https://doi.org/10.1038/s41587-019-0056-8
Dolgin E. First CD123-targeted drug approved after wowing in rare cancer. Nat Biotechnol. 2019 Mar;37(3):202-203. doi: 10.1038/s41587-019-0056-8. PMID: 30833771.
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Showing 1 to 12 of 31 entries