5 Experimental SMA Therapies You Might Find Interesting September 19, 2016September 21, 2016 by admin In Social Clips. 1. AVXS 101 Recently, AveXis’ gene therapy candidate AVXS-101, the only gene therapy for spinal muscular atrophy currently in human testing, has been granted FDA orphan drug status for treatment of all types of SMA. It was also granted FDA breakthrough therapy designation and fast track designation. Read about it and other SMA therapy developments here. Find out more on how AVXS 101 can be used to treat SMA. Print This Page 1 2 3 4 5
