SMArtCAREFollow-up observation and therapy evaluation in spinal muscular atrophy

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.