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| Giugliani,Luciana; Vanzella,Claudia; Zambrano,Marina Bauer; Donis,Karina Carvalho; Wallau,Thaís Klassmann Wendland; Costa,Fernando Machado da; Giugliani,Roberto. |
| Abstract Rare diseases are defined as conditions with a prevalence of no more than 6.5 per 10,000 people. Although each rare disease individually affects a small number of people, collectively, the 6,000 to 8,000 rare conditions (80% of them with genetic cause) affect around 8% of the world’s population. Research about the natural history and underlying pathophysiological mechanisms of rare diseases, as well as clinical trials with new drugs, are important and necessary to develop new strategies for the treatment of these conditions. This report describes the experience of a clinical research group working with rare diseases in a reference center for lysosomal diseases in Brazil (Medical Genetics Service, Hospital de Clínicas de Porto Alegre). The... |
| Tipo: Info:eu-repo/semantics/article |
Palavras-chave: Clinical research; Clinical investigation; Rare diseases; Lysosomal storage diseases; Enzyme replacement therapy. |
| Ano: 2019 |
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572019000200305 |
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| Li,Guowei; Wu,Qianyu; Jin,Yanling; Vanniyasingam,Thuva; Thabane,Lehana. |
| Abstract In general, clinical research network capacity building refers to programs aimed at enhancing networks of researchers to conduct clinical research. Although in the literature there is a large body of research on how to develop and build capacity in clinical research networks, the conceptualizations and implementations remain controversial and challenging. Moreover, the experiences learnt from the past accomplishments and failures can assist in the future capacity building efforts to be more practical, effective and efficient. In this paper, we aim to provide an overview of capacity building in clinical research network by (1) identifying the key barriers to clinical research network capacity building, (2) providing insights into how to overcome... |
| Tipo: Info:eu-repo/semantics/article |
Palavras-chave: Capacity building; Clinical research; Network; Collaboration. |
| Ano: 2018 |
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S1678-91992018000100205 |
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