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doi: 10.1016/j.bpc.2015.03.009 405 GermainABarupalDKLevineSMHansonMR

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Mbah NE, Myers AL, Sajjakulnukit P, et al

Acetyl-L-Carnitin (ALCAR) ist L-Carnitin, welches an eine Acetylgruppe gebunden ist

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This paper presents the first-in-human study of an AAV replacement therapy for infants with SMA-I
