As many of you know, Zolgensma (previously known as AVXS-101) is the proprietary gene therapy candidate from AveXis designed to treat spinal muscular atrophy (SMA). We’ve been covering news regarding this drug on our main website and in our podcasts and flash briefings for the past few months. It’s very likely that the drug will be FDA-approved soon.
As such, we wanted to create a separate sub-forum devoted to Zolgensma. We will be using this forum to post news and updates regarding the treatment, and to talk about what it means for the SMA community as a whole. Though this treatment will only be available to infants with Type I SMA initially, there’s no telling what the future will hold. It’s very possible that it will be made available to adults eventually.
For now, we will be tracking the information and news here. Thanks for joining us, and please feel free to share your thoughts and perspectives regarding Zolgensma.
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 other 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.