AveXis Doses First Patient in Gene Transfer Trial for Spinal Muscular Atrophy

AveXis Doses First Patient in Gene Transfer Trial for Spinal Muscular Atrophy

ChariSMA delivers functional SMN genes to patients using the self-complementary AAV9, which has been proven effective in crossing the bloodbrainbarrier to deliver SMN to motor neurons in preclinical studies. The company expects to improve the

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