Andreea Seferian

Nusinersen in patients older than 7 months with spinal muscular atrophy type 1

Karolina Aragon‐Gawinska, Andreea Seferian, Aurore Daron et al. · Neurology · 2018 · 117 citations

Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial

Laurent Servais, Eugenio Mercuri, Volker Straub et al. · Nucleic Acid Therapeutics · 2021 · 114 citations · Full text

Sitting in patients with spinal muscular atrophy type 1 treated with nusinersen

Karolina Aragon‐Gawinska, Aurore Daron, Ana Ulinici et al. · Developmental Medicine & Child Neurology · 2019 · 46 citations · Full text