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SMA THERAPIES I: P.180AVXS-101 phase 1 gene replacement therapy clinical trial in spinal muscular atrophy type 1: patients treated early with the proposed therapeutic dose were able to sit unassisted at a younger age.
- Source :
-
Neuromuscular Disorders . Oct2018:Supplement 2, Vol. 28, pS82-S82. 1p. - Publication Year :
- 2018
- Subjects :
- *TREATMENT effectiveness
*TREATMENT of spinal muscular atrophy
*GENE therapy
Subjects
Details
- Language :
- English
- ISSN :
- 09608966
- Volume :
- 28
- Database :
- Academic Search Index
- Journal :
- Neuromuscular Disorders
- Publication Type :
- Academic Journal
- Accession number :
- 131609186
- Full Text :
- https://doi.org/10.1016/j.nmd.2018.06.208