SMArtCAREFollow-up observation and therapy evaluation in spinal muscular atrophy

An independent steering committee was elected for the SMArtCARE Registry. The members, some with international expertise, are representatives from neuropediatrics, neurology and the patient initiative "Initiative SMA".

The task of the steering committee is to monitor the progress of the registry from an ethical and scientific point of view. If necessary, the steering committee will make a recommendation to the coordinating Investigator of SMArtCARE to modify or continue the registry. For this purpose, the Steering Committee must be informed about the compliance with the protocol and the corresponding documentation as well as about the patient recruitment.

Furthermore, the steering committee decides in cooperation with the coordinating Investigator of the registry whether data may be made available for a specific scientific question.