The gene therapy drug Zolgensma (AVXS-101) receives FDA approval to treat spinal muscular atrophy in children below 24 months.
AveXis presented data that shows astonishing efficacy of AVXS-101 in SMA type 2 children
TreatSMA anwers the most common questions regarding access to Spinraza treatment
Our official press release on NICE decision to recommend Spinraza
Spinraza® has been recommended as a standard NHS treatment for all patients with spinal muscular atrophy in England and Wales.
#WearYourHurt campaign photos. We are an amazing community!
It was a very interesting and engaging day for the TreatSMA team at the 2019 NICE Conference in Manchester, where NICE and NHS decision makers listened about and acknowledged our concerns.
TreatSMA is talking to NICE and NHS decision makers at a Manchester conference today