Zobrazeno 1 - 9
of 9
pro vyhledávání: '"Jeremy Manuel"'
Publikováno v:
Repositorio Institucional-Ulima
Universidad de Lima
Universidad de Lima
Siendo las calificaciones de riesgo soberano limitantes de decisiones de inversión, intensificadores y moderadores de los ciclos económicos, la presente investigación se centra en entender el comportamiento de los retornos (anormales) del MILA ant
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=od______3883::06c823664385e43d92825bdd6e42a606
https://hdl.handle.net/20.500.12724/15052
https://hdl.handle.net/20.500.12724/15052
Autor:
Maja Di Rocco, Tanya Collin-Histed, Rosella Parini, Carla E. M. Hollak, Ari Zimran, Bruno Bembi, Laura van Dussen, Eugen Mengel, Timothy M. Cox, Helen Michelakakis, Stephan vom Dahl, Pilar Giraldo, Anna Tylki-Szymańska, Johannes M. F. G. Aerts, Nadia Belmatoug, Jeremy Manuel, M. Hrebicek, Miguel Pocovi, Maria Clara Sa Miranda, Jörg Reinke, Patrick Deegan, Yossi Cohen
Publikováno v:
Blood cells, molecules & diseases, 44(1), 41-47. Academic Press Inc.
Gaucher disease is the first lysosomal disorder for which clinically effective enzyme replacement therapy has been introduced. Lifelong treatment with imiglucerase, the recombinant glucocerebrosidase manufactured by the Genzyme Corporation (MA, USA),
Autor:
Jeremy, Manuel, Tanya, Collin-Histed
Publikováno v:
Pediatric endocrinology reviews : PER. 11
The Orphan Drug legislation in the United States and later in the EU has had a significant impact on patients worldwide who suffer from an orphan condition. Apart from providing statutory encouragement and incentives to pharmaceutical companies to de
Publikováno v:
Lysosomal Storage Disorders
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::9a79b9e87e38f6ed5e703c79febc8bdd
https://doi.org/10.1002/9781118514672.ch24
https://doi.org/10.1002/9781118514672.ch24
Publikováno v:
Orphanet Journal of Rare Diseases, 6(1):16. BioMed Central
Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases, BioMed Central, 2011, 6 (1), pp.16. ⟨10.1186/1750-1172-6-16⟩
Orphanet Journal of Rare Diseases, Vol 6, Iss 1, p 16 (2011)
Orphanet Journal of Rare Diseases, 2011, 6 (1), pp.16. ⟨10.1186/1750-1172-6-16⟩
Orphanet Journal of Rare Diseases, 6, 16
Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases, BioMed Central, 2011, 6 (1), pp.16. ⟨10.1186/1750-1172-6-16⟩
Orphanet Journal of Rare Diseases, Vol 6, Iss 1, p 16 (2011)
Orphanet Journal of Rare Diseases, 2011, 6 (1), pp.16. ⟨10.1186/1750-1172-6-16⟩
Orphanet Journal of Rare Diseases, 6, 16
International audience; ABSTRACT: Orphan drugs are often approved under exceptional circumstances, requiring submission of additional data on safety and effectiveness through registries. These registries are mainly focused on one drug only and data i
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2779fe62037f85aba8ef6c007bade7cd
https://dare.uva.nl/personal/pure/en/publications/limitations-of-drug-registries-to-evaluate-orphan-medicinal-products-for-the-treatment-of-lysosomal-storage-disorders(f289412d-6121-4e3d-af2e-3083bc132c44).html
https://dare.uva.nl/personal/pure/en/publications/limitations-of-drug-registries-to-evaluate-orphan-medicinal-products-for-the-treatment-of-lysosomal-storage-disorders(f289412d-6121-4e3d-af2e-3083bc132c44).html
Autor:
Patrick Deegan, Tanya Collin-Histed, Maja Di Rocco, Nadia Belmatoug, Domenica Cappellini, Ari Zimran, Bruno Bembi, Jeremy Manuel, Elena Lukina, Derralynn Hughes, Pilar Giraldo, Olaf Bodamer, Timothy M. Cox, Carla E. M. Hollak, Ashok Vellodi, Helen Michelakakis, Johannes M. F. G. Aerts
Publikováno v:
Blood cells, molecules & diseases, 44(2), 86-87. Academic Press Inc.
Publikováno v:
Orphanet Journal of Rare Diseases
Background EU regulation 726/2004 authorises manufacturers to provide drugs to patients on a temporary basis when marketing authorisation sought centrally for the entire EU is still pending. Individual Member States retain the right to approve and im
Publikováno v:
Orphanet Journal of Rare Diseases
Background Life-saving orphan drugs are some of the most expensive medicines. European Union governments aim to accommodate their provision within stretched healthcare budgets but face pressure to reduce funding of such treatments. Patients struggle