Autori: Bruno C

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Data

SMA-miRs (miR-181a-5p, -324-5p, and -451a) are overexpressed in spinal muscular atrophy skeletal muscle and serum samples

Different trajectories in upper limb and gross motor function in spinal muscular atrophy

Age related treatment effect in type II Spinal Muscular Atrophy pediatric patients treated with nusinersen

Nusinersen in pediatric and adult patients with type III spinal muscular atrophy

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

Type I SMA “new natural history”: long-term data in nusinersen-treated patients

The nonsense mutation stop+4 model correlates with motor changes in Duchenne muscular dystrophy

The Spinal Muscular Atrophy Health Index: Italian validation of a disease-specific outcome measure

Respiratory Trajectories in Type 2 and 3 Spinal Muscular Atrophy in the iSMAC Cohort Study

Clinical Variability in Spinal Muscular Atrophy Type III

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