S61 Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): preliminary pulmonary and ventilatory findings from the phase 3 study (STR1VE)

Shell, Richard; Day, John W.; Chiriboga, CA; Crawford, T. B. B.; Darras, B.; Finkel, R.; Connolly, AM; Iannaccone, ST; Kuntz, N.; Peña, Ldm; Shieh, PB; Smith, EC; Kausar, Imran; Schultz, Megan L.; Feltner, DE; Ogrinc, Francis G.; Macek, Thomas A.; Kernbauer, Elaine; L’Italien, James; Sproule, D.M.; Kaspar, B.; Mendell, J. R.

doi:10.1136/thorax-2019-btsabstracts2019.67

Diagnostic and Therapeutic Advances in Paediatrics 2019

DOI: 10.1136/thorax-2019-btsabstracts2019.67

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S61 Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): preliminary pulmonary and ventilatory findings from the phase 3 study (STR1VE)

Richard Shell

John W. Day

CA Chiriboga

et al.

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S61 Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): preliminary pulmonary and ventilatory findings from the phase 3 study (STR1VE)

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