Browse Items (4 total)

Objective: To validate a useful version of the Motor Function Measure (MFM) in children with neuromuscular diseases aged .90), and discriminant validity was good. Conclusions: The MFM-20 can be used as an outcome measure for assessment of motor…

Spinal muscular atrophy type 1 (SMA-1) is a severe neurodegenerative disorder, which in the absence of curative treatment, leads to death before 1 year of age in most cases. Caring for these short-lived and severely impaired infants requires…

Introduction: This National Hospital Clinical Research Program (PHRC) was conducted in France between 2012 and 2016 to depict palliative practices in spinal muscular atrophy type 1 (SMA-1). New drugs (Nusinersen) have been developed and may modify…
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