Ksenija Gorni

Risdiplam in Type 1 Spinal Muscular Atrophy

Giovanni Baranello, Basil T. Darras, Nicolas Deconinck et al. · New England Journal of Medicine · 2021 · 419 citations · Full text

Medicine, Neurology, Neuroscience +13

Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls

Basil T. Darras, Riccardo Masson, Maria Mazurkiewicz-Bełdzińska et al. · New England Journal of Medicine · 2021 · 274 citations · Full text

Medicine, Neurology, Pediatrics +17

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

Enrico Bertini, Éric Dessaud, Eugenio Mercuri et al. · The Lancet Neurology · 2017 · 114 citations · Full text

Medicine, Pharmacotherapy, Clinical Pharmacology +4

Natural history of Type 2 and 3 spinal muscular atrophy: 2‐year NatHis‐SMA study

M. Annoussamy, A. Seferian, Aurore Daron et al. · Annals of Clinical and Translational Neurology · 2020 · 114 citations · Full text

Medicine, Neurology, Rehabilitation Medicine +17

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, Kristy Rose et al. · The Lancet Neurology · 2022 · 104 citations · Full text

Medicine, Neurology, Pharmacotherapy +6