Miriam Hiebeler

Safety and Treatment Effects of Nusinersen in Longstanding Adult 5q-SMA Type 3 – A Prospective Observational Study

Maggie C. Walter, Stephan Wenninger, Simone Thiele et al. · Journal of Neuromuscular Diseases · 2019 · 218 citations · Full text

Medicine, Neurology, Neuroscience +16

Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophy

Astrid Pechmann, Max Behrens, Katharina Dörnbrack et al. · Brain · 2022 · 70 citations · Full text

Medicine, Neurology, Pediatrics +15

Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study

René Günther, Claudia D. Wurster, Svenja Brakemeier et al. · The Lancet Regional Health - Europe · 2024 · 61 citations · Full text

Medicine, Neurology, Pharmacotherapy +14

Improved upper limb function in non-ambulant children with SMA type 2 and 3 during nusinersen treatment: a prospective 3-years SMArtCARE registry study

Astrid Pechmann, Max Behrens, Katharina Dörnbrack et al. · Orphanet Journal of Rare Diseases · 2022 · 40 citations · Full text

Medicine, Neurology, Pediatrics +14

Methylation of the 4q35 D4Z4 repeat defines disease status in facioscapulohumeral muscular dystrophy

Hannes Erdmann, Florentine Scharf, Stefanie Gehling et al. · Brain · 2022 · 38 citations

Life Sciences, Genetics, Molecular Genetics +15