Ksenija Gorni
Roche (Switzerland)(CH)
Publications by Year
Research Areas
Neurogenetic and Muscular Disorders Research, RNA modifications and cancer, RNA Research and Splicing, Congenital Anomalies and Fetal Surgery, RNA and protein synthesis mechanisms
Most-Cited Works
- → Risdiplam in Type 1 Spinal Muscular Atrophy(2021)395 cited
- → Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls(2021)268 cited
- → Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial(2021)209 cited
- → Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial(2017)114 cited
- → Natural history of Type 2 and 3 spinal muscular atrophy: 2‐year NatHis‐SMA study(2020)111 cited
- → Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial(2022)98 cited
- → Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA)(2023)87 cited
- → 244th ENMC international workshop: Newborn screening in spinal muscular atrophy May 10–12, 2019, Hoofdorp, The Netherlands(2019)69 cited
- → Risdiplam in types 2 and 3 spinal muscular atrophy: A randomised, placebo‐controlled, dose‐finding trial followed by 24 months of treatment(2022)61 cited
- → Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study(2023)53 cited