Shots:

• The partial hold on clinical study for intrathecal administration of AVXS-101 follows the findings of the pre-clinical study demonstrated dorsal root ganglia (DRG) mononuclear cell inflammation accompanied by neuronal cell body degeneration or loss
• The partial hold does not have any impact on marketed Zolgensma or AVXS-101 (IV) clinical studies and will remain available in the US. Novartis will work with the FDA to release the partial hold and resume dosing in the AVXS-101 intrathecal study
• Zolgensma/AVXS-101 is an adeno-associated virus vector-based gene therapy indicated to treat pediatric patients with SMA with bi-allelic mutations in the SMN1 gene in the US

Click here to­ read full press release/ article | Ref: Novartis | Image: Livemint