학술논문
Gene-Replacement Therapy (GRT) in Spinal Muscular Atrophy Type 1 (SMA1): Long-Term Follow-Up From the Onasemnogene Abeparvovec Phase 1/2 Clinical Trial.
Document Type
Article
Author
Mendell, Jerry R.; Shell, Richard; Lehman, Kelly J.; McColly, Markus; Lowes, Linda P.; Alfano, Lindsay N.; Miller, Natalie F.; Iammarino, Megan A.; Church, Kathleen; Ernst, Uwe; Ogrinc, Francis G.; Ouyang, Haojun; Kernbauer, Elaine; Shah, Shivani; L'Italien, James; Sproule, Douglas M.; Feltner, Douglas E.; Al-Zaidy, Samiah
Source
Subject
*SPINAL muscular atrophy
*CLINICAL trials
*
Language
ISSN
0174-304X