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Gene Therapy May Help Babies With Type 1 SMA


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.

Find out more about AVXS-101 gene therapy.

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.

Find out more about the clinical trial for AVXS-101. 

SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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  1. METIN KARAKAS says:

    we have a baby who has SMA type1 and we hope to apply
    AVXS-101 to my sweet daughter KAREN in ANKARA-TURKEY

    • Dianne Roncal, DMD says:

      We are very sorry to hear that, Metin. The best way to know is to seek advice from your doctor. We hope everything turns out well for your dear daughter, Karen. 🙂

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