MacCannell, Drew
Berger, Zdenek http://orcid.org/0000-0001-7987-5683
Kirschner, Janbernd http://orcid.org/0000-0003-1618-7386
Mercuri, Eugenio
Farrar, Michelle A. http://orcid.org/0000-0002-4472-0902
Iannaccone, Susan T.
Kuntz, Nancy L. http://orcid.org/0000-0003-2253-5156
Finkel, Richard S.
Valente, Marta
Muntoni, Francesco http://orcid.org/0000-0002-9102-5232
Funding for this research was provided by:
Biogen
Article History
Accepted: 11 January 2022
First Online: 26 January 2022
Declarations
:
: This study was sponsored by Biogen. The open access fee was funded by Biogen.
: Drew MacCannell, Zdenek Berger, and Marta Valente are full-time employees of and hold stock/stock options in Biogen. Janbernd Kirschner received funding for consultancy, educational, and research activities concerning SMA from AveXis/Novartis Gene Therapies, Biogen, Roche, and Scholar Rock. Eugenio Mercuri has participated in advisory boards for SMA studies for AveXis/Novartis Gene Therapies, Biogen, Ionis, Novartis, and Roche; is a principal investigator for ongoing Biogen/Ionis and Roche clinical trials; and has received funding from Famiglie SMA Italy, Italian Telethon, and SMA Europe. Michelle A. Farrar has participated in advisory boards for and received honoraria from Biogen, Roche, and Novartis. Susan T. Iannaccone receives partial salary support from the following grants: the Muscular Dystrophy Association, NeuroNEXT U24NS107176, NIH Wellstone Muscular Dystrophy Center P50HD087351, and Taysha Gene Therapies; receives research support from CureSMA, DoD W81XWH2010293, Parent Project for MD, and industry (Biogen, FibroGen, Novartis, PTC, ReveraGen, Sarepta, and Scholar Rock); and has served on medical advisory boards for Biogen, Novartis, and Sarepta. Nancy L. Kuntz has participated in advisory boards for argenx, Audentes, AveXis/Novartis Gene Therapies, Biogen, Cytokinetics, PTC, Roche, and Sarepta, and received honoraria from Biogen and clinical research funding direct to her institution from Audentes, AveXis/Novartis Gene Therapies, Biogen, and Sarepta. Richard S. Finkel has participated in SMA advisory boards for AveXis/Novartis Gene Therapies, Biogen, Roche, and Scholar Rock; received honoraria from AveXis/Novartis Gene Therapies, Biogen, Elsevier, Excerpta Medica, Roche, and Voyager; received research funding to institution from Biogen and Ionis for conduct of the CS3A, CHERISH, ENDEAR, NURTURE, and SHINE studies, and from AveXis/Novartis Gene Therapies, Cytokinetics, Roche, and Scholar Rock; was a member of the data safety monitoring boards for the AveXis AVX-101 phase I gene transfer study and the Roche Moonfish phase Ib study; serves in an advisory capacity for the nonprofit organizations Cure SMA, SMA Europe, SMA Foundation, and SMA Reach (UK); and receives royalty payments from the Children’s Hospital of Philadelphia for licensing fees obtained for use of the CHOP INTEND motor function scale. Francesco Muntoni has participated in advisory boards for Biogen, Dyne Therapeutics, and Pfizer; has been a consultant for AveXis/Novartis Gene Therapies, Biogen, Roche, Santhera, and Sarepta; received grants from the European Commission, Muscular Dystrophy Association USA, Muscular Dystrophy UK, and Medical Research Council; received research funding/investigator grants from Biogen and Sarepta; and has been a principal investigator for Ionis nusinersen trials, the Roche olesoxime and risdiplam trials in SMA, and the Avexis/Novartis gene therapy trial of onasemnogene abeparvovec.
: The nusinersen clinical trials were conducted in compliance with the Declaration of Helsinki, the International Council for Harmonisation Good Clinical Practice guidelines, and local regulatory requirements.
: Written informed consent was obtained from the parent or legal guardian and the participant, as required by local guidelines and age, for each clinical trial.
: Not applicable.
: Requests for data supporting this manuscript should be submitted to the Biogen Clinical Data Request Portal (ExternalRef removed).
: Not applicable.
: JK, EM, MAF, STI, NLK, RSF, and FM participated in the collection of data. DM, ZB, and MV participated in the study design and DM conducted the pharmacokinetic analyses. DM, ZB, JK, EM, MAF, STI, NLK, RSF, MV, and FM participated in the analysis and interpretation of data and in drafting and critically revising the manuscript. All authors have read and approved the final submitted paper and agree to be accountable for the work.