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Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results

Document Type

Article

Author

Mendell, J.R.; Lowes, L.P.; Alfano, L.N.; Lehman, K.J.; Church, K.; Reash, N.F.; Iammarino, M.A.; Sabo, B.; Pozsgai, E.R.; Lewis, S.; Griffin, D.A.; Potter, R.; Neuhaus, S.; Li, X.; Stevenson, H.; Rodino-Klapac, L.R.

Source

In: Nature Medicine. (Nature Medicine, January 2024, 30(1):199-206)

Subject

Language

English

ISSN

1546170X
10788956

Online Access

Full Text (Nature Journals) Web of Science JCR 저널정보 Scopus Find it@PNU

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