Raggruppamento Secondario: Atrofia Muscolare Spinale (SMA)

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Data

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

Longitudinal natural history of type I spinal muscular atrophy: a critical review

Evaluation of body composition as a potential biomarker in spinal muscular atrophy

Respiratory Needs in Patients with Type 1 Spinal Muscular Atrophy Treatedÿwith Nusinersen

A critical review of patient and parent caregiver oriented tools to assess health-related quality of life, activity of daily living and caregiver burden in spinal muscular atrophy

Circulating MyomiRs as Potential Biomarkers to Monitor Response to Nusinersen in Pediatric SMA Patients

Intrathecal administration of nusinersen in adult and adolescent patients with spinal muscular atrophy and scoliosis: Transforaminal versus conventional approach

Development of an academic disease registry for spinal muscular atrophy

Nusinersen in type 1 spinal muscular atrophy: Twelve-month real-world data

Long-term progression in type II spinal muscular atrophy: A retrospective observational study

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