A gene therapy may be able to help babies with type 1 spinal muscular atrophy (SMA). AveXis has published the results of the first phase of a clinical trial for its drug AVXS-101 which aims to deliver a working copy of the SMN gene to the patient’s motor neuron cells, overriding the child’s own faulty copy of the SMN2 gene and enabling the restoration of motor neuron function.
The gene will be delivered via a harmless virus which can cross the blood-brain barrier to reach its target. The clinical trial was conducted with 15 babies—newborns to six months old—with type 1 SMA. They each received an injection of the virus, with 12 of the babies receiving a high dose of AVXS-101 and three receiving a lower dose.
All 12 babies who received the high dose reached 13.6 months without any disease events. Eleven of the babies achieved head control, nine could sit unaided for five seconds, seven for at least ten seconds and five for 30 seconds. Two of the babies could walk independently.
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