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Avxs 101

A new trial of gene therapy in SMA announced

Today Avexis announced the start of a new clinical trial of gene therapy in SMA.

The trial will be conducted in the United States and enroll at least 15 babies with SMA type 1. The babies will each receive one intravenous injection of AVXS-101 and will be closely followed for at least 14 months.

This will be a pivotal trial which means its results may form the basis of drug authorisation application.

Avexis also said they are working towards starting a trial of AVXS-101 in patients with SMA type 2.

Complete press release can be read here.

We are excited to see that this new therapy for SMA is making headways so fast!

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