SMArtCAREFollow-up observation and therapy evaluation in spinal muscular atrophy

17.09.2025

WARNING: Due to maintenance, documentation for SMArtCARE will not be possible on 25th September 2025 from 3.00 to 5.00 pm. Please make sure to log off sufficiently in advance in order to avoid loss of data. Please do not log in again before the data management gives the clear-all.

04.12.2024

Newsletter #4

07.10.2024

Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study (Weiß C et al., The Lancet Regional Health, 2024)

19.09.2024

Anwendungsbegleitende Datenerhebung to the application of Risdiplam starts on October 30, 2024 (Pressemitteilung des G-BA)

08.04.2024

• Clinical Effectiveness of Newborn Screening for Spinal Muscular Atrophy: A Nonrandomized Controlled Trial (Schartz O et al, JAMA Pediatr, 2024)

06.02.2024

• Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study (Günther R et al., The Lancet Regional Health, 2024)

27.02.2024

• 5qSMA: standardised retrospective natural history assessment in 268 patients with four copies of SMN2 (Vill K et al., Journal of Neurology, 2024)

07.06.2023

The new website www.smartcare.de goes live.