1. Natural history of Type 2 and 3 spinal muscular atrophy: 2‐year NatHis‐SMA study. Issue 2 (24th December 2020) Authors: Annoussamy, Mélanie; Seferian, Andreea M.; Daron, Aurore; Péréon, Yann; Cances, Claude; Vuillerot, Carole; De Waele, Liesbeth; Laugel, Vincent; Schara, Ulrike; Gidaro, Teresa; Lilien, Charlotte; Hogrel, Jean‐Yves; Carlier, Pierre; Fournier, Emmanuel; Lowes, Linda; Gorni, Ksenija; Ly‐Le Moal, Myri... Journal: Annals of clinical and translational neurology Issue: Volume 8:Issue 2(2021) Page Start: 359 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial. Issue 10 (October 2021) Authors: Mercuri, Eugenio; Muntoni, Francesco; Baranello, Giovanni; Masson, Riccardo; Boespflug-Tanguy, Odile; Bruno, Claudio; Corti, Stefania; Daron, Aurore; Deconinck, Nicolas; Servais, Laurent; Straub, Volker; Ouyang, Haojun; Chand, Deepa; Tauscher-Wisniewski, Sitra; Mendonca, Nuno; Lavrov, Arseniy; Se... Journal: Lancet neurology Issue: Volume 20:Issue 10(2021) Page Start: 832 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. Scoring People With Spinal Muscular Atrophy on the Motor Function Measure Using the Microsoft Kinect. Issue 1 (25th October 2022) Authors: Vincent-Genod, Dominique; Rippert, Pascal; Coton, Justine; Le Goff, Laure; Barriere, Aurélie; Berruyer, Anne; Bernard, Marjorie; Garde, Camille; Gutierrez-Garcia, Marta; Gilabert, Stéphanie; Gomes-Lisboade-Souza, Adriana; Daron, Aurore; Servais, Laurent; Thomann, Guillaume; Vuillerot, Carole Journal: Pediatric physical therapy Issue: Volume 35:Issue 1(2023) Page Start: 36 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
7. Sitting in patients with spinal muscular atrophy type 1 treated with nusinersen. (4th December 2019) Authors: Aragon‐Gawinska, Karolina; Daron, Aurore; Ulinici, Ana; Vanden Brande, Laura; Seferian, Andreea; Gidaro, Teresa; Scoto, Mariacristina; Deconinck, Nicolas; Servais, Laurent Other Names: Benezit Audrey investigator.; Mathieu Marie‐Laure investigator.; Cances Claude investigator.; Durigneux Julien investigator.; Ropars Juliette investigator.; Chouchane Mondher investigator.; Forey Peggy investigator.; Lazaro Leila investigator.; Hughes Imelda investigator.; Illingworth Marjorie in... Journal: Developmental medicine & child neurology Issue: Volume 62:Number 3(2020) Page Start: 310 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
8. Therapeutic interventions for spinal muscular atrophy: preclinical and early clinical development opportunities. (4th May 2021) Authors: Servais, Laurent; Baranello, Giovanni; Scoto, Mariacristina; Daron, Aurore; Oskoui, Maryam Journal: Expert opinion on investigational drugs Issue: Volume 30:Number 5(2021) Page Start: 519 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
9. X-linked myotubular myopathy: A prospective international natural history study. (16th April 2019) Authors: Annoussamy, Mélanie; Lilien, Charlotte; Gidaro, Teresa; Gargaun, Elena; Chê, Virginie; Schara, Ulrike; Gangfuß, Andrea; D'Amico, Adele; Dowling, James J.; Darras, Basil T.; Daron, Aurore; Hernandez, Arturo; de Lattre, Capucine; Arnal, Jean-Michel; Mayer, Michèle; Cuisset, Jean-Marie; Vuillerot, C... Journal: Neurology Issue: Volume 92:Number 16(2019) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗