Patient reported outcome measures in spinal muscular atrophy and duchenne muscular dystrophy: review of instruments and their inclusion in clinical and regulatory processes
Despite the availability of PROMs, ObsROMs, and CROMs for SMA and DMD, their use in clinical trials and regulatory documents is limited and inconsistent. Greater standardization and systematic inclusion of these measures are needed to support patient-centered drug development and evaluation.
Authors
- O. Ciani
- Francesco Malandrini (ORCID: https://orcid.org/0000-0002-0245-9849)
- Clarissa Spataro (ORCID: https://orcid.org/0000-0002-6202-1267)
- Valeria Sansone
- Adele D’amico
- Michela Meregaglia
- Annalisa Scopinaro
Institutions
- Université Paris-Saclay (FR)
- Federazione Nazionale Imprese Elettroniche ed Elettrotecniche (IT)
- Institut des Sciences des Plantes de Paris Saclay (FR)
- Centro Clinico Nemo (IT)
- Roche (Italy) (IT)
- Bambino Gesù Children's Hospital (IT)
- Bocconi University (IT)
Publication Details
- Journal
- Neurological Sciences
- Published
- 2026-02-13
- DOI
- https://doi.org/10.1007/s10072-025-08600-1
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Roche Italia