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doi: 10.3390/cancers17213524 99 KrestensenKKHendriksTFEGrgicADerweduweMDe SmetFHeerenRMAet al
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Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: interim efficacy and safety results from the phase 2 NURTURE study
A., Leijten-van de Gevel, I
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