Upper limb progression in Duchenne muscular dystrophy: Insights from a 36-month longitudinal study using the PUL 20
A total of 219 patients provided 684 paired 36-month assessments. Ambulatory status significantly affected total, shoulder, elbow, and distal scores at baseline. The largest 36-month decline in total scores was found in the 58 transitioning patients (11.62 points, 95%CI = -12.40, 10.84), followed by non-ambulant and ambulant subgroups (n = 116 and n = 86 respectively). The largest declines were seen in patients with baseline entry score of 4 (-11.97, 95% CI = -13.48, -10.46) and 5 (-11.55, 95% CI = -12.46, -10.63), with smaller declines for other entry scores.ConclusionsThe 36-month analysis confirms a clear trend of functional decline across time points, with the transitioning group exhibiting the greatest changes in upper limb function. These findings provide valuable insights for designing trials and offer a reference for long-term comparison of treatment efficacy in both experimental and real-world setting.
Authors
- G. De Luca (ORCID: https://orcid.org/0000-0002-2747-7250)
- Riccardo Masson (ORCID: https://orcid.org/0000-0002-9311-452X)
- Elena Pegoraro (ORCID: https://orcid.org/0000-0002-7740-4156)
- Adele D’Amico (ORCID: https://orcid.org/0000-0003-2438-2624)
- S Paolucci
- Valeria Sansone (ORCID: https://orcid.org/0000-0002-1354-3800)
- Claudia Dosi (ORCID: https://orcid.org/0000-0001-7591-9124)
- Giorgia Coratti (ORCID: https://orcid.org/0000-0001-6666-5628)
- Chiara Panicucci (ORCID: https://orcid.org/0000-0003-4571-611X)
- Enrica Rolle
- Tiziana Mongini (ORCID: https://orcid.org/0000-0003-4061-4261)
- Federica Ricci (ORCID: https://orcid.org/0000-0003-2019-612X)
- Ilaria Cavallina
- Vincenzo Nigro (ORCID: https://orcid.org/0000-0002-3378-5006)
- Bianca Buchignani (ORCID: https://orcid.org/0000-0003-0954-5384)
- Eugenio Maria Mercuri (ORCID: https://orcid.org/0000-0002-9851-5365)
- Anna Capasso (ORCID: https://orcid.org/0000-0003-4061-4501)
- Roberta Battini (ORCID: https://orcid.org/0000-0002-7889-1355)
- Marika Pane (ORCID: https://orcid.org/0000-0002-4851-6124)
- Antonella Pini (ORCID: https://orcid.org/0000-0003-3603-5478)
- Luca Bello (ORCID: https://orcid.org/0000-0002-3075-6525)
- Angela Lucia Berardinelli (ORCID: https://orcid.org/0000-0002-4645-8055)
- Melania Giannotta
- Silvia Frosini (ORCID: https://orcid.org/0000-0001-6169-4382)
- Emilio Albamonte (ORCID: https://orcid.org/0000-0002-8465-4460)
- Michela Catteruccia (ORCID: https://orcid.org/0000-0003-0785-5004)
- Riccardo Zanin (ORCID: https://orcid.org/0000-0002-6200-3632)
- Riccardo Not
- Alice Gardani
- Claudio Bruno
- Esther Picillo
- Gianpaolo Cicala
Institutions
- Università Cattolica del Sacro Cuore (IT)
- University of Pisa (IT)
- Istituto Giannina Gaslini (IT)
- Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda (IT)
- Centro Clinico Nemo (IT)
- Institute of Neurological Sciences (IT)
- Bambino Gesù Children's Hospital (IT)
- University of Campania "Luigi Vanvitelli" (IT)
- Fondazione Stella Maris (IT)
- Telethon Institute Of Genetics And Medicine (IT)
- Fondazione IRCCS Istituto Neurologico Carlo Besta (IT)
- University of Turin (IT)
Publication Details
- Journal
- Journal of Neuromuscular Diseases
- Published
- 2025-09-18
- DOI
- https://doi.org/10.1177/22143602251355318
- Citations
- 1
- Primary Topic
- Muscle Physiology and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.81