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Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease.
Hollak CE, vom Dahl S, Aerts JM, Belmatoug N, Bembi B, Cohen Y, Collin-Histed T, Deegan P, van Dussen L, Giraldo P, Mengel E, Michelakakis H, Manuel J, Hrebicek M, Parini R, Reinke J, di Rocco M, Pocovi M, Sa Miranda MC, Tylki-Szymanska A, Zimran A, Cox TM. Hollak CE, et al. Among authors: manuel j. Blood Cells Mol Dis. 2010 Jan 15;44(1):41-7. doi: 10.1016/j.bcmd.2009.09.006. Epub 2009 Oct 4. Blood Cells Mol Dis. 2010. PMID: 19804996
Guidelines for the restart of imiglucerase in patients with Gaucher disease: recommendations from the European Working Group on Gaucher disease.
Hollak CE, Aerts JM, Belmatoug N, Bembi B, Bodamer O, Cappellini D, Collin-Histed T, Cox TM, Deegan P, Giraldo P, Hughes D, Lukina E, Manuel J, Michelakakis H, Di Rocco M, Vellodi A, Zimran A. Hollak CE, et al. Among authors: manuel j. Blood Cells Mol Dis. 2010 Mar-Apr;44(2):86-7. doi: 10.1016/j.bcmd.2009.12.002. Blood Cells Mol Dis. 2010. PMID: 20022271 No abstract available.
Compassionate use of orphan drugs.
Hyry HI, Manuel J, Cox TM, Roos JC. Hyry HI, et al. Among authors: manuel j. Orphanet J Rare Dis. 2015 Aug 21;10:100. doi: 10.1186/s13023-015-0306-x. Orphanet J Rare Dis. 2015. PMID: 26292942 Free PMC article.
The legal imperative for treating rare disorders.
Hyry HI, Roos JC, Manuel J, Cox TM. Hyry HI, et al. Among authors: manuel j. Orphanet J Rare Dis. 2013 Sep 6;8:135. doi: 10.1186/1750-1172-8-135. Orphanet J Rare Dis. 2013. PMID: 24010951 Free PMC article.
Protocol for a randomised controlled trial of ketamine versus ketamine and behavioural activation therapy for adults with treatment-resistant depression in the community.
Beaglehole B, Porter R, Douglas K, Lacey CJ, de Bie A, Jordan J, Mentzel C, Thwaites B, Manuel J, Murray G, Frampton C, Glue P. Beaglehole B, et al. Among authors: manuel j. BMJ Open. 2024 May 1;14(5):e084844. doi: 10.1136/bmjopen-2024-084844. BMJ Open. 2024. PMID: 38692731 Free PMC article. Clinical Trial.
366 results