Zolgensma Helps Mobility of SMA Children in Qatari Real-world Study

Zolgensma Helps Mobility of SMA Children in Qatari Real-world Study

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Zolgensma Helps Mobility of SMA Children in Qatari Real-world Study

The gene therapy Zolgensma safely improved mobility in young children with spinal muscular atrophy (SMA) living in Qatar, a real-world study shows. The case series study, “Gene therapy for spinal muscular atrophy: the Qatari experience,” was published in the journal Nature Gene Therapy. Zolgensma is an approved gene therapy developed by AveXis, now part of Novartis, for the treatment of SMA. It uses a harmless adeno-associated virus vector to deliver a functional copy of the SMN1 gene…

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