Article
Prospective observational cohort studies for studying rare diseases: the European PedNet Haemophilia Registry.
Haemophilia : the official journal of the World Federation of Hemophilia - 1 Jul 2014
Fischer K, Ljung R, Platokouki H, Liesner R, Claeyssens S, Smink E, van den Berg H M
Abstract excerpt
Haemophilia is a rare disease. To improve knowledge, prospective studies of large numbers of subjects are needed. To establish a large well-documented birth cohort of patients with haemophilia enabling studies on early presentation, side effects and outcome of treatment. Twenty-one haemophilia treatment centres have been collecting data on all children with haemophilia with FVIII/IX levels up to 25% born from...
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