Iannaccone, Susan T. http://orcid.org/0000-0003-0851-3516
Cai, Chunyu
Rhem, Brittney
Batley, Kaitlin
Rajaram, Veena
Greenberg, Benjamin M.
Dharia, Sachi
Zaidman, Craig M.
Funding for this research was provided by:
Sarepta Therapeutics
Article History
Received: 21 March 2024
Revised: 30 April 2024
Accepted: 8 May 2024
First Online: 22 June 2024
Declarations
:
: STI receives research support from industry (Elpida, Novartis, Biogen, Sarepta Therapeutics, PTC Therapeutics, Scholar Rock, FibroGen, RegenxBio, and ReveraGen) and the Department of Defense W81XWH2010293, Parent Project Muscular Dystrophy, and Cure SMA. She has served on medical advisory boards for Novartis, Biogen, Genentech, and Sarepta Therapeutics. She receives partial salary support from the following grants: National Institutes of Health Wellstone Muscular Dystrophy Center P50HD087351, NeuroNEXT U24NS107176, and the Muscular Dystrophy Association. CC, BR, and VR declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. KB receives research support from industry (Elpida, Novartis, Biogen, Sarepta Therapeutics, PTC Therapeutics, Scholar Rock, FibroGen, Regenxbio, NS Pharma, and ReveraGen) and Cure SMA. She has served on medical advisory boards for Biogen, Reata Pharmaceuticals, PTC Therapeutics, UCB, Pfizer, Catalyst, and Sarepta Therapeutics. BMG has received consulting fees from Alexion, Novartis, EMD Serono, Horizon Therapeutics, Genentech/Roche, Signant Health, IQVIA, Sandoz, Genzyme, Immunovant, TG Therapeutics, Cycle Pharmaceuticals, Partnership for Health Analytic Research, InterVenn, Arialys Therapeutics, Bayer, Janssen, Clene, Syneos Health, and PRIME Education. He has received grant funding from Regeneron Pharmaceuticals, Anokion, and National Institutes of Health. He has equity in Clene and GenrAb. He receives royalties from UpToDate. He serves as an unpaid member of the board of the Siegel Rare Neuroimmune Association. SD is an employee of Sarepta Therapeutics and may have stock options. CMZ receives research support from Biogen and Novartis, receives speaker fees from Sarepta Therapeutics and Chugai, and serves on an advisory board for Sarepta Therapeutics.
: Ethical review and approval were not required for this case report in accordance with the local legislation and institution requirements. The patient’s guardian provided written informed consent for the publication of any potentially identifiable images or data included in this article.