1. D.08 ACT DMD (Ataluren Confirmatory Trial in Duchenne Muscular Dystrophy): effect of Ataluren on timed function tests (TFT) in nonsense mutation (nm) DMD. (17th June 2016) Authors: Goemans, N; Campbell, C; McDonald, CM; Voit, T; Luo, X; Elfring, G; Kroger, H; Riebling, P; Ong, T; Spiegel, R; Peltz, SW; Bushby, K Journal: Canadian journal of neurological sciences Issue: Volume 43(2016)Supplement 2 Page Start: S15 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. P.063 SUNFISH Part 1 results and Part 2 trial design in patients with type 2/3 spinal muscular atrophy (SMA) receiving risdiplam (RG7916). (5th June 2019) Authors: Campbell, C; Mercuri, E; Baranello, G; Kirschner, J; Servais, L; Goemans, N; Pera, M; Buchbjerg, J; Yeung, W; Kletzl, H; Gerber, M; Czech, C; Cleary, Y; Gorni, K; Khwaja, O Journal: Canadian journal of neurological sciences Issue: Volume 46(2019)Supplement 1 Page Start: S31 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. P.063 SUNFISH Part 1 results and Part 2 trial design in patients with type 2/3 spinal muscular atrophy (SMA) receiving risdiplam (RG7916). (June 2019) Authors: Campbell, C; Mercuri, E; Baranello, G; Kirschner, J; Servais, L; Goemans, N; Pera, M; Buchbjerg, J; Yeung, W; Kletzl, H; Gerber, M; Czech, C; Cleary, Y; Gorni, K; Khwaja, O Journal: Canadian journal of neurological sciences Issue: Volume 46(2019)Supplement 1 Page Start: S31 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
4. Relationships between motor impairments and activity limitations in patients with neuromuscular disorders. Issue 3 (23rd October 2008) Authors: Vandervelde, L; Van den Bergh, P Y K; Renders, A; Goemans, N; Thonnard, J-L Journal: Journal of neurology, neurosurgery and psychiatry Issue: Volume 80:Issue 3(2009) Page Start: 326 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
5. Unusual molecular findings in autosomal recessive spinal muscular atrophy. Issue 6 (June 1996) Authors: Matthijs, G; Schollen, E; Legius, E; Devriendt, K; Goemans, N; Kayserili, H; Apäk, M Y; Cassiman, J J Journal: Journal of medical genetics Issue: Volume 33:Issue 6(1996) Page Start: 469 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗