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Patient reported outcome measures in spinal muscular atrophy and duchenne muscular dystrophy: review of instruments and their inclusion in clinical and regulatory processes

Crossref DOI link: https://doi.org/10.1007/s10072-025-08600-1

Published Online: 2026-02-13

Published Print: 2026-03

Update policy: https://doi.org/10.1007/springer_crossmark_policy

Authors

Malandrini, Francesco https://orcid.org/0000-0002-0245-9849
Spataro, Clarissa

Meregaglia, Michela

Sansone, Valeria

D’amico, Adele

Scopinaro, Annalisa

Ciani, Oriana
Funding

Funding for this research was provided by:

Università Commerciale Luigi Bocconi
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Text and Data Mining valid from 2026-02-13

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Article History

Received: 7 September 2025

Accepted: 2 December 2025

First Online: 13 February 2026

Declarations

:

: Francesco Malandrini, Michela Meregaglia, Oriana Ciani, and Annalisa Scopinaro declare that they have no competing interests. Clarissa Spataro is an employee of Roche. Adele D’Amico has received fees in the last two years for communication activities at congresses and participation in scientific boards for Novartis, Roche, and Astellas. Valeria Sansone provides intellectual and scientific advice for Novartis, Roche, Biogen, Lupin, PTC, Santhera, Dyne, Avidity, Verthex, Arthex, Sanofi, and Pepgen, and receives honoraria for participation in advisory boards.

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