Programma MD-PhD della Scuola di Medicina

Innovative treatments for children and adolescents with Duchenne muscular dystrophy (DMD) and long-term course of illness: Instructor prof. Benedetto Vitiello

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Programma

Research activities include:

1. a) A multicenter, observational study of patients receiving Translarna based on inclusion of their data in a registry (STRIDE, Strategic Targeting of Registries and International Database of Excellence, coordinator for Italy: Prof. Eugenio Mercuri, Catholic University, Rome).

This study is intended to enroll 200 patients across ~50 care centers in Europe and other regions over a period of ~ 2 years.

1. b) A multi-centre, double-blind, parallel group, 36-60 month study, comparing three corticosteroid regimens (FOR-DMD; coordinator: Prof. Robert Griggs, Rochester University, New York). This clinical trial addresses the pragmatic hypothesis that daily corticosteroids (prednisone or deflazacort) will be of greater benefit in terms of function and subject/parent satisfaction than intermittent corticosteroids (prednisone).
2. c) Long-term natural history of DMD (GUP15011N Telethon Multicentric; coordinator: Prof. Eugenio Mercuri, Catholic University, Rome). This study will provide a unique opportunity to identify prognostic factors related to loss of ambulation, which is the most important endpoint in the age range considered in this project.

Note

Progetto del corso: Patologie del sistema motorio - Malattie neuro-muscolari.

Ospedale Regina Margherita ella Citta della Scienza e della Salute di Torino 

Scientific qualification of the PI: Entrez PubMed Link to publications of prof Vitiello

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