D’Amore, Simona
Mckie, Mark
Fahey, Andrew
Bleloch, David
Grillo, Giuseppina
Hughes, Michael
Ramaswami, Uma http://orcid.org/0000-0002-4703-7447
Funding for this research was provided by:
Society for Mucopolysaccharide Disease (MPS Society), UK (Society of Mucopolysaccharide Disease, UK)
Amicus Therapeutics, UK
Article History
Received: 28 June 2023
Accepted: 15 December 2023
First Online: 11 January 2024
Declarations
:
: This project was conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki, and that are consistent with Good Clinical Practice and all applicable regulatory requirements. As it was a quality improvement project on monitoring clinical symptoms, it did not require ethics approval.
: No restrictions.
: SD: none related to this manuscript. SD is currently employed at Chiesi Farmaceutici S.p.A., unrelated to this project. MM none. DB is a patient and MH and AF are employees of Health Touch Ltd. GG none. UR: none related to this manuscript. UR has received research grants from Amicus, Takeda, Intrabio, Sanofi; Advisory board and lecture fees from Amicus, Takeda, Sanofi.