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Authors
O’Sullivan, Fiona
Tomazos, Ioannis https://orcid.org/0000-0003-0290-4583
van Spronsen, Francjan J.
Szabo, Shelagh M.
Venkataraman, Maanasa
Huria, Lavanya
Smith, Neil
Molony, Lachlan
Ingalls, Kim
Somera-Molina, Kathleen
Zhang, Rongrong
Harding, Cary O.
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Funding
Funding for this research was provided by:
PTC Therapeutics
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Article History
Received: 14 January 2025
Accepted: 22 May 2025
First Online: 20 June 2025
Declarations
:
: Not applicable.
: Not applicable.
: FOS, SMS, MV, and LH are employees of Broadstreet HEOR, which received a contract from PTC Therapeutics Inc. for the conduct of this review. IT, NS, LM, KI, KSM* and RZ are employees of PTC Therapeutics Inc. and may hold stock in the company. Francjan J. van Spronsen declares that his institution received consulting fees for scientific advisory board meetings; grants for clinical and non-clinical studies; honoraria for speaking engagements and clinical trial funding from BioMarin; and has received fees for advisory board meetings from Agios, alltRNA, Arla Food Int, Applied Pharma Research, Eurocept, Illumina, LogicBio, Lucane, Moderna, Nestle-Codexis Alliance, Nutricia, Origin Biosciences, and Orphan Europe; grants from Alexion, BioMarin, ESPKU, NPKUA, NPKUV, Nutricia, PKU Research Foundation (NL), Sobi, and the Tyrosinemia Foundation; and consulting fees from Applied Pharma Research, BioMarin, Nutricia, Orphan Europe, and Pluvia Biotech. COH has received consulting fees from BioMarin, Jnana Therapeutics, PTC Therapeutics, Sanofi, Pfizer, Synlogic, Tessera Therapeutics, and Tome Biosciences and conducted clinical research trials for BioMarin, Jnana Therapeutics, Sanofi, and Synlogic related to treatment of PAH deficiency. *at the time of study completion.
