1. 188 SUNFISH 3-year efficacy and safety of risdiplam in types 2 and 3 SMA. Issue 9 (12th August 2022) Authors: Servais, Laurent; Day, John W; Mazzone, Elena S; Nascimento, Andres; Oskoui, Maryam; Baranello, Giovanni; Gerber, Marianne; Martin, Carmen; Yeung, Wai Yin; Mercuri, Eugenio Journal: Journal of neurology, neurosurgery and psychiatry Issue: Volume 93:Issue 9(2022) Page Start: e2 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Bayesian adaptive dose‐escalation designs for simultaneously estimating the optimal and maximum safe dose based on safety and efficacy. (9th July 2017) Authors: Yeung, Wai Yin; Reigner, Bruno; Beyer, Ulrich; Diack, Cheikh; Sabanés bové, Daniel; Palermo, Giuseppe; Jaki, Thomas Journal: Pharmaceutical statistics Issue: Volume 16:Number 6(2017) Page Start: 396 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Bayesian adaptive dose‐escalation procedures for binary and continuous responses utilizing a gain function. (9th September 2015) Authors: Yeung, Wai Yin; Whitehead, John; Reigner, Bruno; Beyer, Ulrich; Diack, Cheikh; Jaki, Thomas Journal: Pharmaceutical statistics Issue: Volume 14:Number 6(2015:Nov./Dec.) Page Start: 479 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) ↗