Autori: Day JW

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Data

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

Risdiplam in Type 1 Spinal Muscular Atrophy

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

2-Year Change in Revised Hammersmith Scale Scores in a Large Cohort of Untreated Paediatric Type 2 and 3 SMA Participants

Revised upper limb module for spinal muscular atrophy: 12?month changes

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

Quantitative Evaluation of Lower Extremity Joint Contractures in Spinal Muscular Atrophy: Implications for Motor Function

Revised upper limb module for spinal muscular atrophy: Development of a new module

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