SMArtCAREFollow-up observation and therapy evaluation in spinal muscular atrophy

New therapeutic options for treating patients with spinal muscular atrophy (SMA) bring opportunities, but also new challenges.

With nusinersen, risdiplam and gene therapy (onasemnogen abeparvovec), there are now three different drugs on the market for the treatment of SMA. However, the approval of these drugs is predominantly based on clinical trials in children and to date there is little experience of efficacy and safety outside of these population or over a longer observation period.

In order to better assess the therapeutic effects of the currently approved drugs, it is necessary to systematically collect and evaluate real-world data from untreated and treated patients with SMA over a long period of time in the context of routine clinical practice. Therefore, in 2017, the disease-specific SMArtCARE Registry was established in a joint initiative of neuropaediatricians, neurologists and the patient organization "Initiative SMA" of the German Society for Muscular Dystrophy (DGM) e.V. for the German-speaking region (Germany/Austria/Switzerland). The SMArtCARE registry is distinct from, and in addition to, the Treat-NMD Registry, in which patients can self-register.

Currently, SMArtCARE relies on financial support from the pharmaceutical industry. Nevertheless, full data ownership rests with the SMArtCARE network. The industry has no influence on the design of the registry or on the analysis and interpretation of the data. To ensure the scientific independence of the SMArtCARE registry, no data are shared directly with pharmaceutical companies to fulfil regulatory requirements, but instead are sent to independent EU-based institution for analysis.

The data collection has been approved by the relevant authorities and ethics committees.