1. Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy. Issue 3 (15th February 2022) Authors: Pane, Marika; Coratti, Giorgia; Pera, Maria Carmela; Sansone, Valeria A.; Messina, Sonia; d'Amico, Adele; Bruno, Claudio; Salmin, Francesca; Albamonte, Emilio; De Sanctis, Roberto; Sframeli, Maria; Di Bella, Vincenzo; Morando, Simone; Palermo, Concetta; Frongia, Anna Lia; Antonaci, Laura; Capasso... Journal: Annals of clinical and translational neurology Issue: Volume 9:Issue 3(2022) Page Start: 404 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Nusinersen in pediatric and adult patients with type III spinal muscular atrophy. Issue 8 (24th June 2021) Authors: Pera, Maria Carmela; Coratti, Giorgia; Bovis, Francesca; Pane, Marika; Pasternak, Amy; Montes, Jacqueline; Sansone, Valeria A.; Dunaway Young, Sally; Duong, Tina; Messina, Sonia; Mizzoni, Irene; D'Amico, Adele; Civitello, Matthew; Glanzman, Allan M.; Bruno, Claudio; Salmin, Francesca; Morando, Si... Journal: Annals of clinical and translational neurology Issue: Volume 8:Issue 8(2021) Page Start: 1622 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. 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. Issue 1 (January 2022) Authors: Mercuri, Eugenio; Deconinck, Nicolas; Mazzone, Elena S; Nascimento, Andres; Oskoui, Maryam; Saito, Kayoko; Vuillerot, Carole; Baranello, Giovanni; Boespflug-Tanguy, Odile; Goemans, Nathalie; Kirschner, Janbernd; Kostera-Pruszczyk, Anna; Servais, Laurent; Gerber, Marianne; Gorni, Ksenija; Khwaja, ... Journal: Lancet neurology Issue: Volume 21:Issue 1(2022) Page Start: 42 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
4. 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. Issue 1 (January 2022) Authors: Mercuri, Eugenio; Deconinck, Nicolas; Mazzone, Elena S; Nascimento, Andres; Oskoui, Maryam; Saito, Kayoko; Vuillerot, Carole; Baranello, Giovanni; Boespflug-Tanguy, Odile; Goemans, Nathalie; Kirschner, Janbernd; Kostera-Pruszczyk, Anna; Servais, Laurent; Gerber, Marianne; Gorni, Ksenija; Khwaja, ... Journal: Lancet neurology Issue: Volume 21:Issue 1(2022) Page Start: 42 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
5. 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. Issue 1 (January 2022) Authors: Mercuri, Eugenio; Deconinck, Nicolas; Mazzone, Elena S; Nascimento, Andres; Oskoui, Maryam; Saito, Kayoko; Vuillerot, Carole; Baranello, Giovanni; Boespflug-Tanguy, Odile; Goemans, Nathalie; Kirschner, Janbernd; Kostera-Pruszczyk, Anna; Servais, Laurent; Gerber, Marianne; Gorni, Ksenija; Khwaja, ... Journal: Lancet neurology Issue: Volume 21:Issue 1(2022) Page Start: 42 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
6. Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial. Issue 12 (December 2022) Authors: Masson, Riccardo; Mazurkiewicz-Bełdzińska, Maria; Rose, Kristy; Servais, Laurent; Xiong, Hui; Zanoteli, Edmar; Baranello, Giovanni; Bruno, Claudio; Day, John W; Deconinck, Nicolas; Klein, Andrea; Mercuri, Eugenio; Vlodavets, Dmitry; Wang, Yi; Dodman, Angela; El-Khairi, Muna; Gorni, Ksenija; Jaber... Journal: Lancet neurology Issue: Volume 21:Issue 12(2022) Page Start: 1110 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
7. The SPTLC1 p.S331 mutation bridges sensory neuropathy and motor neuron disease and has implications for treatment. (10th August 2022) Authors: Fiorillo, Chiara; Capodivento, Giovanna; Geroldi, Alessandro; Tozza, Stefano; Moroni, Isabella; Mohassel, Payam; Cataldi, Matteo; Campana, Chiara; Morando, Simone; Panicucci, Chiara; Pedemonte, Marina; Brolatti, Noemi; Siliquini, Sabrina; Traverso, Monica; Baratto, Serena; Debellis, Doriana; Magr... Journal: Neuropathology & applied neurobiology Issue: Volume 48:Number 7(2022) Page Start: n/a Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗