AveXis Gene Therapy Used in Series of SMA Clinical Trials Both Safe and ‘Transformative,’ Executives Say in Interview – SMA News Today
SMA News Today |
AveXis Gene Therapy Used in Series of SMA Clinical Trials Both Safe and 'Transformative,' Executives Say in Interview
SMA News Today The Penn study found severe liver and motor neuron toxicity, respectively, in the three monkeys and three piglets it treated at high dose with an AAVhu68 vector used to deliver a gene therapy. That vector is a variant of the AAV9 vectors being used in … |
