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| Official Name | Description | Author |
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COMMISSION STAFF WORKING DOCUMENT Summary of the Impact Assessment on Rare Diseases: Europe's challenges. Impact assessment
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On this basis, the preferred option is to bring forward proposals for a Community strategy for rare [...]
| European Commission |
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COMMISSION STAFF WORKING DOCUMENT Proposal for a COUNCIL RECOMMENDATION on a European action in the field of rare diseases. Impact assessment
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There is a need to act in a cohesive manner, as there is no effective way in which the Member States [...]
| European Commission |
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Expanded newborn screening in Europe 2007
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By January 2007 seven European countries had expanded, and more are considering the expansion of the [...]
| O. A. Bodamer & G. F. Hoffmann & M. Lindner J Inherit Metab Dis (2007) 30:439–444 |
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Clinical trials of orphan medicines
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The development of orphan medicines presents many challenges, the main being to obtain suffi cient e [...]
| Brendan M Buckley |
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Empowerment of patients: lessons from the rare diseases community Lancet 2008; 371: 2048–51
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In most developed countries, the emergence and empowerment of patients’ organisations began wi [...]
| Ségolène Aymé, Anna Kole, Stephen Groft |
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Gene therapy of inherited diseases The Lancet Vol 371
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Thousands of rare diseases are caused by a Mendelian genetic error. So far, more than 1800 genes ass [...]
| Alain Fischer, Marina Cavazzana-Calvo |
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Making rare diseases a public-health and research priority/Direct-to-consumer advertising under a diff erent name The Lancet Vol 371
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Making rare diseases a public-health and research priority/Direct-to-consumer advertising under a di [...]
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Does orphan drug legislation really answer the needs of patients? Lancet 2008; 371: 2041–44
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Orphan-drug legislation (panel)1 was intended to make drugs for rare diseases suffi ciently profi ta [...]
| Marlene E Haff ner, Josep Torrent-Farnell, Paul D Maher |
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Rare diseases: what’s next? The Lancet Vol 371
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Rare diseases are also called health orphans—for most of them there is little knowledge of cau [...]
| Giuseppe Remuzzi, Silvio Garattini Clinical Research Centre for Rare Diseases ‘Aldo e Cele Daccò’, Mario Negri Institute for Pharmacological Research, Laboratori Negri Bergamo, 24125 Bergamo, Italy |
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Why rare diseases are an important medical and social issue The Lancet Vol 371
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Rare diseases are an important public-health issue and a challenge for the medical community. They a [...]
| Arrigo Schieppati, Jan-Inge Henter, Erica Daina, Anita Aperia |
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