Article
Characterization of the DMD/BMD patient population in Czech Republic and Slovakia using an innovative registry approach.
Neuromuscular disorders : NMD - 1 Apr 2009
Brabec Petr, Vondrácek Petr, Klimes Daniel, Baumeister Sarah, Lochmüller Hanns, Pavlík Tomás, Gregor Jakub
Abstract excerpt
Effective planning of clinical trials requires an appropriate number of patients who fulfil given inclusion criteria. In the case of so called "orphan" diseases, such as Duchenne and Becker muscular dystrophy (DMD/BMD), the number of suitable patients within one country is usually limited. We developed a detailed registry of Czech and Slovak DMD/BMD patients which may contribute to cooperation on the European...
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