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Authors
Lah, Melissa https://orcid.org/0000-0002-2406-8961
Kopesky, Jessica https://orcid.org/0009-0008-5030-683X
Vucko, Erika https://orcid.org/0000-0003-0143-9675
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Funding
Funding for this research was provided by:
PTC Therapeutics MP, Inc.
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Article History
First Online: 6 June 2026
Declarations
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: Melissa Lah has served on advisory boards for Applied Therapeutics, BioMarin Pharmaceutical, Maze Therapeutics, and PTC Therapeutics; and serves as a principal investigator on studies with BioMarin Pharmaceutical and PTC Therapeutics. Jessica Kopesky has received consulting funding for advisory boards and/or speaking engagements from Acer Therapeutics, BioMarin Pharmaceutical, Cycle Pharmaceuticals, Horizon Therapeutics, Mead Johnson Nutrition, Moderna, Nutricia, PTC Therapeutics, Relief Therapeutics, and Ultragenyx. Erika Vucko has received consulting funding for advisory boards and/or speaking engagements for advisory boards for BioMarin Pharmaceutical, PTC Therapeutics, Horizon/Amgen, and Acer Therapeutics; and serves as a principal investigator on studies with BioMarin Pharmaceutical, Otsuka, and Agios.
: This article is based on previously conducted studies, all of which were conducted with the appropriate ethics committee approvals.
