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Authors
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Funding
Funding for this research was provided by:
BioMarin Pharmaceutical
Sanofi Genzyme
Swedish Orphan Biovitrum
Takeda
Ultragenyx Pharmaceutical
Alexion Pharmaceuticals
Amicus Therapeutics
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Article History
Received: 2 December 2023
Accepted: 14 July 2024
First Online: 22 July 2024
Declarations
:
: Not applicable.
: Not applicable.
: Lucy McKay is an employee of Medics4RareDiseases. Mr Dan Jeffries is a Medics4RareDiseases trustee.
