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Article History

Received: 8 March 2017

Accepted: 9 May 2017

First Online: 31 May 2017

Competing interests

: FM is involved as principal investigator in the following clinical trials: nusinersen (SHINE, sponsored by Ionis and Biogen); olesoxime (OLEOS, sponsored by Roche). He has participated in scientific advisory board activities for Roche; Biogen and Avexis, and is also a member of the Pfizer rare disease scientific advisory board. MS is involved as sub-investigator in SHINE clinical trial and is principal investigator in OLEOS clinical trial. RFS is involved as principal investigator in the following SMA clinical trials: nusinersen (CS3A, ENDEAR, CHERISH, NURTURE and SHINE, sponsored by Ionis and Biogen) and CK-2127107 (CY 5021 study, sponsored by Cytokinetics and Astellas). He has participated in scientific advisory board activities for Ionis, Biogen, Roche, Novartis and AveXis; has served on the DSMB for the Roche RG7800 and AveXis AVXS-101 phase 1 study; and has served as an advisor to CureSMA (US), the SMA Foundation (US), SMA REACH (UK) and SMA Europe. EM is involved as principal investigator in the following clinical trials: nusinersen (SHINE, sponsored by Ionis and Biogen); olesoxime (OLEOS, sponsored by Roche). He has participated in scientific advisory board activities for Ionis, Roche; Biogen and Avexis.