1. Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study. Issue 2 (21st January 2016) Authors: Kolb, Stephen J.; Coffey, Christopher S.; Yankey, Jon W.; Krosschell, Kristin; Arnold, W. David; Rutkove, Seward B.; Swoboda, Kathryn J.; Reyna, Sandra P.; Sakonju, Ai; Darras, Basil T.; Shell, Richard; Kuntz, Nancy; Castro, Diana; Iannaccone, Susan T.; Parsons, Julie; Connolly, Anne M.; Chiribog... Journal: Annals of clinical and translational neurology Issue: Volume 3:Issue 2(2016) Page Start: 132 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Natural history of infantile‐onset spinal muscular atrophy. Issue 6 (8th December 2017) Authors: Kolb, Stephen J.; Coffey, Christopher S.; Yankey, Jon W.; Krosschell, Kristin; Arnold, W. David; Rutkove, Seward B.; Swoboda, Kathryn J.; Reyna, Sandra P.; Sakonju, Ai; Darras, Basil T.; Shell, Richard; Kuntz, Nancy; Castro, Diana; Parsons, Julie; Connolly, Anne M.; Chiriboga, Claudia A.; McDonal... Journal: Annals of neurology Issue: Volume 82:Issue 6(2017) Page Start: 883 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy. Issue 8 (July 2016) Authors: Straub, Volker; Balabanov, Pavel; Bushby, Kate; Ensini, Monica; Goemans, Nathalie; De Luca, Annamaria; Pereda, Alejandra; Hemmings, Robert; Campion, Giles; Kaye, Edward; Arechavala-Gomeza, Virginia; Goyenvalle, Aurelie; Niks, Erik; Veldhuizen, Olav; Furlong, Pat; Stoyanova-Beninska, Violeta; Wood... Journal: Lancet neurology Issue: Volume 15:Issue 8(2016:Jul.) Page Start: 882 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗