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AVXS-101 Gene Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update

B26. CLINICAL STUDIES IN PEDIATRIC SLEEP, HOME VENTILATION AND NEUROMUSCULAR DISEASE Pub Date : 2019-05-01 DOI:10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a2789
R. Shell, J. W. Day, C. Chiriboga, Thomas O. Crawford, Basil T. Darras, Richard S. Finkel, Anne M. Connolly, S. Iannaccone, Nancy L Kuntz, L. Pena, M. Schultz, Perry B. Shieh, E. Smith, D. Feltner, F. Ogrinc, Thomas A. Macek, C. Wells, L. Muehring, J. L'italien, D. Sproule, Brian K. Kaspar, J. Mendell
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AVXS-101基因替代疗法(GRT)治疗1型脊髓性肌萎缩症(SMA1):关键的3期研究(STR1VE)更新
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B26. CLINICAL STUDIES IN PEDIATRIC SLEEP, HOME VENTILATION AND NEUROMUSCULAR DISEASE
B26. CLINICAL STUDIES IN PEDIATRIC SLEEP, HOME VENTILATION AND NEUROMUSCULAR DISEASE
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