Dati del progetto
|
Principal Investigator |
Dr.ssa Silvia Bonanno |
|
Ente ospitante |
Fondazione IRCCS Istituto Neurologico ‘C. Besta’ |
|
Area Tematica |
Neurologia |
|
Data di inizio Progetto |
24/02/2025 |
|
Data di fine Progetto |
23/02/2028 |
|
Importo assegnato |
500.000,00 € |
|
Tipo Progetto |
Individuale |
PROJECT AIM
Spinal muscular atrophy (SMA) natural history is changing with the advent of disease-modifying drugs (DMDs). However, SMA patients’ response to DMDs is partial and highly variable, with marked benefits in the precocious treatments. This project aims to:
•Discover non-invasive BMs, providing predictive, prognostic and pharmacodynamic information, to promptly address patients to the most suited therapeutic intervention.
•Identify specific SMA patients’ subclasses-omic signatures, to disclose tailored therapeutic targets of innovative treatments complementary to DMDs.
•Test the efficacy of a novel nanotechnology-based treatment targeting atrophy in a patient specific skeletal muscle in vitro model to overcome muscle impairment and contribute to MN functionality.