Arianna Iosca
Motor assessment in DMD: introducing a Novel Parameter usable after LoA
Autori
- ARIANNA IOSCA (DEPARTMENT OF BRAIN AND BEHAVIORAL SCIENCES, UNIVERSITY OF PAVIA, PAVIA, ITALY – RESIDENT IN NEUROPSICHIATRIA INFANTILE)
- ALICE GARDANI (DEPARTMENT OF CHILD NEUROLOGY AND PSYCHIATRY, IRCCS C. MONDINO FOUNDATION, PAVIA, ITALY – NEURODEVELOPMENTAL THERAPIST (TNPEE))
- SARA FUSCO (DEPARTMENT OF BRAIN AND BEHAVIORAL SCIENCES, UNIVERSITY OF PAVIA, PAVIA, ITALY – RESIDENT IN NEUROPSICHIATRIA INFANTILE)
- MARIA IRENE DAINESI (DEPARTMENT OF BRAIN AND BEHAVIORAL SCIENCES, UNIVERSITY OF PAVIA, PAVIA, ITALY – RESIDENT IN NEUROPSICHIATRIA INFANTILE)
- LAURA CARRARO (DEPARTMENT OF BRAIN AND BEHAVIORAL SCIENCES, UNIVERSITY OF PAVIA, PAVIA, ITALY – RESIDENT IN NEUROPSICHIATRIA INFANTILE)
- CARLO ALBERTO QUARANTA (DEPARTMENT OF BRAIN AND BEHAVIORAL SCIENCES, UNIVERSITY OF PAVIA, PAVIA, ITALY – RESIDENT IN NEUROPSICHIATRIA INFANTILE)
- STEFANO PARRAVICINI (DEPARTMENT OF BRAIN AND BEHAVIORAL SCIENCES, UNIVERSITY OF PAVIA, PAVIA, ITALY, DEPARTMENT OF CHILD NEUROLOGY AND PSYCHIATRY, IRCCS C. MONDINO FOUNDATION, PAVIA, ITALY – NEUROPSICHIATRA INFANTILE)
- ANGELA LUCIA BERARDINELLI (DEPARTMENT OF CHILD NEUROLOGY AND PSYCHIATRY, IRCCS C. MONDINO FOUNDATION, PAVIA, ITALY – NEUROPSICHIATRA INFANTILE)
Presentatore
ARIANNA IOSCA (DEPARTMENT OF BRAIN AND BEHAVIORAL SCIENCES, UNIVERSITY OF PAVIA, PAVIA, ITALY)
Modalità
Poster Session
Abstract
“Duchenne muscular dystrophy (DMD) is characterized by progressive muscle degeneration, resulting in progressive weakness and severe motor disability, with cardiorespiratory involvement.
The forthcoming therapies press for finding adequate outcome measures for the different stages of the disease.
Timed functional tests (TFT) are common clinical trial endpoints in Duchenne muscular dystrophy (DMD) and have been used to explore and better define the natural history of DMD. After the loss of ambulation (LoA) is more difficult to identify the changes in motor function and its association with vital parameters. Upper limb motor function is usually evaluated by PUL.
The present study aims to describe the timed motor performance of moving from a supine to prone position (STP) and its association to age, TFT, motor function tests (NSSA, PUL) and respiratory parameters (vital capacity CV).
We enrolled 22 patients between the ages of 5 and 17 years.
The results indicate a correlation between STP and the motor variables used, while the data analysis with respiratory parameters did not yield any significant results.
STP seems a straightforward and quick TFT, suitable for use even post-LoA. Further data are needed to confirm its validity and usefulness in clinical practice.”