1. A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy. (24th October 2017) Authors: Victor, Ronald G.; Sweeney, H. Lee; Finkel, Richard; McDonald, Craig M.; Byrne, Barry; Eagle, Michelle; Goemans, Nathalie; Vandenborne, Krista; Dubrovsky, Alberto L.; Topaloglu, Haluk; Miceli, M. Carrie; Furlong, Pat; Landry, John; Elashoff, Robert; Cox, David Journal: Neurology Issue: Volume 89:Number 17(2017) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Issue 10101 (23rd September 2017) Authors: McDonald, Craig M; Campbell, Craig; Torricelli, Ricardo Erazo; Finkel, Richard S; Flanigan, Kevin M; Goemans, Nathalie; Heydemann, Peter; Kaminska, Anna; Kirschner, Janbernd; Muntoni, Francesco; Osorio, Andrés Nascimento; Schara, Ulrike; Sejersen, Thomas; Shieh, Perry B; Sweeney, H Lee; Topaloglu... Journal: Lancet Issue: Volume 390:Issue 10101(2017) Page Start: 1489 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Ataluren treatment of patients with nonsense mutation dystrophinopathy. Issue 4 (October 2014) Authors: Bushby, Katharine; Finkel, Richard; Wong, Brenda; Barohn, Richard; Campbell, Craig; Comi, Giacomo P.; Connolly, Anne M.; Day, John W.; Flanigan, Kevin M.; Goemans, Nathalie; Jones, Kristi J.; Mercuri, Eugenio; Quinlivan, Ros; Renfroe, James B.; Russman, Barry; Ryan, Monique M.; Tulinius, Mar; Voi... Journal: Muscle & nerve Issue: Volume 50:Issue 4(2014:Oct.) Page Start: 477 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
4. Clinical Variability in Spinal Muscular Atrophy Type III. Issue 6 (2nd October 2020) Authors: Coratti, Giorgia; Messina, Sonia; Lucibello, Simona; Pera, Maria Carmela; Montes, Jacqueline; Pasternak, Amy; Bovis, Francesca; Exposito Escudero, Jessica; Mazzone, Elena Stacy; Mayhew, Anna; Glanzman, Allan M.; Young, Sally Dunaway; Salazar, Rachel; Duong, Tina; Muni Lofra, Robert; De Sanctis, R... Journal: Annals of neurology Issue: Volume 88:Issue 6(2020) Page Start: 1109 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
5. Effects of glucocorticoids and idebenone on respiratory function in patients with duchenne muscular dystrophy. Issue 9 (5th November 2012) Authors: Buyse, Gunnar M.; Goemans, Nathalie; van den Hauwe, Marleen; Meier, Thomas Journal: Pediatric pulmonology Issue: Volume 48:Issue 9(2013:Sep.) Page Start: 912 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
6. Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial. Issue 9979 (2nd May 2015) Authors: Buyse, Gunnar M; Voit, Thomas; Schara, Ulrike; Straathof, Chiara S M; D'Angelo, M Grazia; Bernert, Günther; Cuisset, Jean-Marie; Finkel, Richard S; Goemans, Nathalie; McDonald, Craig M; Rummey, Christian; Meier, Thomas Journal: Lancet Issue: Volume 385:Issue 9979(2015) Page Start: 1748 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
7. Emerging therapies for Duchenne muscular dystrophy. Issue 9 (September 2022) Authors: Markati, Theodora; Oskoui, Maryam; Farrar, Michelle A; Duong, Tina; Goemans, Nathalie; Servais, Laurent Journal: Lancet neurology Issue: Volume 21:Issue 9(2022) Page Start: 814 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
8. European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy. (September 2020) Authors: Kirschner, Janbernd; Butoianu, Nina; Goemans, Nathalie; Haberlova, Jana; Kostera-Pruszczyk, Anna; Mercuri, Eugenio; van der Pol, W. Ludo; Quijano-Roy, Susana; Sejersen, Thomas; Tizzano, Eduardo F.; Ziegler, Andreas; Servais, Laurent; Muntoni, Francesco Journal: European journal of paediatric neurology Issue: Volume 28(2020) Page Start: 38 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
9. Gene therapy for spinal muscular atrophy: hope and caution. Issue 4 (April 2021) Authors: Goemans, Nathalie Journal: Lancet neurology Issue: Volume 20:Issue 4(2021) Page Start: 251 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
10. How glucocorticoids change life in Duchenne muscular dystrophy. Issue 10119 (3rd February 2018) Authors: Goemans, Nathalie Journal: Lancet Issue: Volume 391:Issue 10119(2018) Page Start: 406 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗