1. A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier. Issue 1 (16th November 2018) Authors: Sturm, Stefan; Günther, Andreas; Jaber, Birgit; Jordan, Paul; Al Kotbi, Nada; Parkar, Nikhat; Cleary, Yumi; Frances, Nicolas; Bergauer, Tobias; Heinig, Katja; Kletzl, Heidemarie; Marquet, Anne; Ratni, Hasane; Poirier, Agnès; Müller, Lutz; Czech, Christian; Khwaja, Omar Journal: British journal of clinical pharmacology Issue: Volume 85:Issue 1(2019) Page Start: 181 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Longitudinal characterization of biomarkers for spinal muscular atrophy. Issue 5 (11th April 2017) Authors: Bonati, Ulrike; Holiga, Štefan; Hellbach, Nicole; Risterucci, Céline; Bergauer, Tobias; Tang, Wakana; Hafner, Patricia; Thoeni, Alain; Bieri, Oliver; Gerlach, Irene; Marquet, Anne; Khwaja, Omar; Sambataro, Fabio; Bertolino, Alessandro; Dukart, Juergen; Fischmann, Arne; Fischer, Dirk; Czech, Chris... Journal: Annals of clinical and translational neurology Issue: Volume 4:Issue 5(2017) Page Start: 292 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Oral SMN2 splicing modifiers in spinal muscular atrophy: Proof-of-mechanism and ongoing clinical studies. (June 2017) Authors: Marquet, Anne; Sturm, Stefan; Kletzl, Heidemarie; Günther, Andreas; Czech, Christian; Poirier, Agnes; Seabrook, Timothy; Müller, Lutz; Kinch, Russell; Ratni, Hasane; Gorni, Ksenija; Metzger, Friedrich; Khwaja, Omar Journal: European journal of paediatric neurology Issue: Volume 21(2017)Supplement 1 Page Start: e226 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 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) ↗