Action plan 2011-2013

3 - Care and trial site registry - CTSRBiobanks


1 -

Ensure quality and quantity of data in the CTSR

Develop further the quality of data and range of information and disease types represented by the CTSR. Introduce systematic updates to all information.

2 -

Service offers, infrastructure

Provide disease specific expertise for trial selection. Develop a core dataset to allow data exchange between other rare disease registries and the CTSR

Achievements 2011-2013

With 310 registered centers, the CTSR now contains 50% more sites than in January 2011. Five official requests from industry and two from academia for site identification, often in combination with TREAT-NMD patient registry information, were received between 2010 and 2013. In addition, the CTSR was used to analyse the current situation for Duchenne muscular dystrophy patients within the EU-funded CARE-NMD project. Therefore, the CTSR has become quite a valuable resource for industry and academia in the field of neuromuscular diseases.

In 2013, the CTSR was expanded to cover twelve more diseases and now encompasses 32 rare diseases. These newly introduced diseases belong mostly to the neurodegenerative field that is the object of research in the NeurOmics project.  Already 40 sites have entered data concerning these diseases.

In January 2014, the CTSR introduced a new facility: the PhenoSearch, which allows researchers to put out a call to registered centers for patients fitting a particular clinical profile. It is hoped that this will speed up the identification of new disease causing mutations and better stratification of patients.


Sunil Rodger, Hanns Lochmüller, Adrian Tassoni, Kathrin Gramsch, Kirsten König, Kate Bushby, Volker Straub, Rudolf Korinthenberg and Janbernd Kirschner, The TREAT-NMD care and trial site registry: an online registry to facilitate clinical research for neuromuscular diseases. Orphanet Journal of rare Diseases,

12 Apr 2017