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SMA - TREATMENT: EP.274 Onasemnogene Abeparvovec Gene Therapy in Presymptomatic spinal muscular atrophy (SMA): SPR1NT study update in children with 3 Copies of SMN2.
- Source :
-
Neuromuscular Disorders . 2021 Supplement 1, Vol. 31, pS133-S133. 1p. - Publication Year :
- 2021
- Subjects :
- *SPINAL muscular atrophy
*GENE therapy
Subjects
Details
- Language :
- English
- ISSN :
- 09608966
- Volume :
- 31
- Database :
- Academic Search Index
- Journal :
- Neuromuscular Disorders
- Publication Type :
- Academic Journal
- Accession number :
- 152517331
- Full Text :
- https://doi.org/10.1016/j.nmd.2021.07.299