Journal: Plos One

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Age, corticosteroid treatment and site of mutations affect motor functional changes in young boys with Duchenne Muscular Dystrophy

Predictive models in SMA II natural history trajectories using machine learning: A proof of concept study

North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up

Diagnostic journey in Spinal Muscular Atrophy: Is it still an odyssey?

Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53

Ambulatory function in spinal muscular atrophy: Age-related patterns of progression

Upper limb function in Duchenne muscular dystrophy: 24 month longitudinal data

Functional levels and MRI patterns of muscle involvement in upper limbs in Duchenne muscular dystrophy

Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment tool

A randomized clinical trial in preterm infants on the effects of a home-based early intervention with the ‘CareToy System’.

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