The Integrated Approach in Patients with Spinal Muscular Atrophy in the Era of Early Diagnosis, Etiopathogenic Therapies and Multidisciplinary Standards of Care and Rehabilitation Interventions Leads to New Phenotypes
These findings reveal that the development of new SMA phenotypes is closely linked to the stage of disease at which treatment is initiated. Earlier intervention consistently enables patients to acquire previously unattainable motor skills. Consequently, enhancing diagnostic precision and expediting therapy initiation is crucial for maximizing clinical benefits and facilitating optimal functional outcomes.
Authors
- Andrada Mirea (ORCID: https://orcid.org/0009-0008-0532-2021)
- Georgiana Nicolae (ORCID: https://orcid.org/0000-0001-6028-2407)
- Mădălina Cristina Leanca
- Andrei Căpitănescu (ORCID: https://orcid.org/0000-0003-2183-0727)
- Gelu Onose (ORCID: https://orcid.org/0000-0002-6346-8941)
- Constantin Munteanu (ORCID: https://orcid.org/0000-0002-1084-7710)
Institutions
- Carol Davila University of Medicine and Pharmacy (RO)
- Clinical Emergency Hospital Bucharest (RO)
- Grigore T. Popa University of Medicine and Pharmacy (RO)
- Spitalului Clinic de Urgență pentru Copii Maria Sklodowska Curie (RO)
Publication Details
- Journal
- Life
- Published
- 2025-11-10
- DOI
- https://doi.org/10.3390/life15111731
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00