The natural history of spinal muscular atrophy (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. Since 2017 nusinersen, risdiplam and gene therapy (onasemnogene abeparvovec) have been approved for the treatment of patients with SMA. The approval of these orphan drugs is often based on clinical trial data from a small subgroup of paediatric SMA patients. Thus, additional systematic data collection is essential to evaluate safety and effectiveness of these treatments over longer observation periods and in a broader spectrum of patients.

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

SMArtCARE is a joint initiative of neurologists, paediatricians, and patients’ organisations. 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 has been developed to collect data on SMA patients from their health-care providers. Data are collected during routine patient visits and items for data collection are aligned with the international consensus for SMA registries.

Different pharmaceutical companies provide financial support for this registry. However, the contractual basis has been regulated in such a way that data collection and interpretation are independent of these companies.

We have 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 centres and is contractually guaranteed.
If you are interested in participating in the SMArtCARE data collection, please contact us: SMArtCARE@uniklinik-freiburg.de. The data will be collected at your centre. If the corresponding ethical approval and consent of the patients are available, they can be entered into the central database.

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.