The natural history of SMA changed over the last decade due to changes in care and the development of targeted treatments for SMA.

Targeted treatments for SMA have been developed in preclinical and clinical studies. Among these, Nusinersen is the first drug to be approved in the United States and in Europe since 2016 and 2017 for the treatment of SMA. The approval of nusinersen for all types of SMA is based on well-controlled clinical trial data of a small subgroup of pediatric SMA patients. Thus, a systematical data capture is crucial to evaluate outcomes and the effect of treatment after prolonged treatment periods and in a broader spectrum of patients.

In Germany, there is already an existing registry for patients with spinal muscular atrophy. Please, indicate this opportunity to your patients (www.sma-register.de).

SMArtCARE is a joint initiative of neurologists, child neurologists, and patients’ organizations. The aim of SMArtCARE is to collect longitudinal “real-world data” on all available SMA patients independent of their actual treatment regime as disease-specific SMA registry. For this purpose, an online platform for SMA patients will be provided seen by health-care providers in German-speaking countries. Data are collected during routine patient visits and items for data collection are aligned with the international consensus for SMA registries.

Biogen provides financial support for this registry. In the future, further support from other companies is conceivable to secure the long-term sustainability of the registry. However, the contractual basis has been regulated in such a way that data collection and interpretation are independent of pharmaceutical companies.

We have already developed recommendations for the clinical evaluation of patients with SMA and provide corresponding documentation sheets in the download area. Compensation is provided for the documentation efforts of the participating centers and is contractually guaranteed. If you are interested in participating in the SMArtCARE data collection, please contact us: SMArtCARE@uniklinik-freiburg.de. The data would first be collected at your center. If the corresponding ethical approval and consent of the patients are available, the transfer to the central data collection is possible later.

SMArtCARE is a network for clinical research in spinal muscular atrophy. If you have your own research ideas or would like to validate new evaluation tools, please contact us. You may be able to use the infrastructure of SMArtCARE for your project, thus avoiding unnecessary extra work.