1. 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) ↗
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). (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.064 FIREFISH Part 1: 1-year results on motor function in infants with Type 1 spinal muscular atrophy (SMA) receiving risdiplam (RG7916). (June 2019) Authors: Seabrook, TJ; Baranello, G; Servais, L; Day, JW; Deconinck, N; Mercuri, E; Klein, A; Darras, B; Masson, R; Kletzl, H; Cleary, Y; El-Khairi, M; Czech, C; Gerber, M; Nguyen, C; Gelblin, K; Gorni, K; Khwaja, O; Cabalteja, C 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. 074 Long-term effect of idebenone in reducing respiratory function decline in patients with Duchenne muscular dystrophy. Issue 6 (27th May 2022) Authors: Servais, L; Mayer, OH; McDonald, CM; Voit, T; Mercuri, E; Buyse, GM Journal: Journal of neurology, neurosurgery and psychiatry Issue: Volume 93:Issue 6(2022) Page Start: A35 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
5. P.064 FIREFISH Part 1: 1-year results on motor function in infants with Type 1 spinal muscular atrophy (SMA) receiving risdiplam (RG7916). (5th June 2019) Authors: Seabrook, TJ; Baranello, G; Servais, L; Day, JW; Deconinck, N; Mercuri, E; Klein, A; Darras, B; Masson, R; Kletzl, H; Cleary, Y; El-Khairi, M; Czech, C; Gerber, M; Nguyen, C; Gelblin, K; Gorni, K; Khwaja, O; Cabalteja, C 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) ↗