Progetto FRRB ID 4956734 - Spinal muscular atrophy translating customized health innovations to newborn and grown-up patients (SMA-TCHING)
Dati del progetto
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Principal Investigator |
Dr.ssa Silvia Bonanno |
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Ente ospitante |
Fondazione IRCCS Istituto Neurologico "C. Besta" |
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Area Tematica |
Neurologia |
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Data di inizio Progetto |
24/02/2025 |
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Data di fine Progetto |
23/02/2028 |
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Importo assegnato |
500.000,00 € |
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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.