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AGIL-AADC Gene Therapy Results in Sustained Improvements in Motor and Developmental Milestones over 5 Years in Children with AADC Deficiency
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Effect of ataluren on age at loss of ambulation in nonsense mutation Duchenne muscular dystrophy: observational data from the STRIDE Registry
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Ataluren in patients aged ≥ 2 to < 5 years with nonsense mutation Duchenne muscular dystrophy (nmDMD): 28-week results from a phase 2 study
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