Treat-nmd Outcome Measures

This project aimed to consolidate the activities that the Italian clinical researchers coordinated within the European network project TREAT-NMD (EU-FP6 program) regarding the development of new functional measures in patients with Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA).
In particular, natural history data collection at 24 months was completed for DMD using measures developed with TREAT-NMD (6-minute walk test, 6MWT; North Star functional scale). The data collected on Italian patients were compared with those collected by European partners (Belgium and UK).
For SMA, the European data collection using 6MWT in SMA type 3 was completed, providing natural history data for ambulatory patients, and a study was carried out on the possible cardiac involvement in patients with SMA.
A new functional measure for the upper limbs in non-ambulatory patients has also been developed for both pathologies.
The activities entailed also the organization of dedicated workshops with the participation of patient organizations and European and American clinical colleagues  to compare the results in the different patient cohorts and reach a consensus on the most suitable and meaningful functional measures for patients’ evaluation in clinical trials and for the routine follow up.