Contact details
Katharina Dörnbrack
Main
Janbernd Kirschner
Alternate
Publications
Data source publications
- SMArtCARE - A platform to collect real-life outcome data of patients with spinal muscular atrophy(Pechmann A et al., Orphanet Journal of Rare Diseases, 2019)
- Zur Gentherapie der Spinalen Muskelatrophie mit Onasemnogene Abeparvovec. Stellungnahme der Gesellschaft für Neuropädiatrie
- Development and Pilot Test of the Registry Evaluation and Quality Standards Tool: An Information Technology-Based Tool to Support and Review Registries
- 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
- Clinical Effectiveness of Newborn Screening for Spinal Muscular Atrophy: A Nonrandomized Controlled Trial.
- 5qSMA: standardised retrospective natural history assessment in 268 patients with four copies of SMN2. 5qSMA: standardised retrospective natural history assessment in 268 patients with four copies of SMN2.
- Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study.
- Improvements in Walking Distance during Nusinersen Treatment - A Prospective 3-year SMArtCARE Registry Study.
- Improved upper limb function in non-ambulanSMA type 2 and 3 during nusinersen treatment: a prospective 3-years SMArtCARE registry study.
- Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophy.