Author: Andi Thornton
FDA approves Zolgensma, the first gene replacement therapy for SMA
The gene therapy drug Zolgensma (AVXS-101) receives FDA approval to treat spinal muscular atrophy in children below 24 months.
Gene therapy works wonders in children with SMA type 2
AveXis presented data that shows astonishing efficacy of AVXS-101 in SMA type 2 children
Questions and answers about Spinraza in the UK
TreatSMA anwers the most common questions regarding access to Spinraza treatment
Press release on NICE decision to recommend Spinraza treatment
Our official press release on NICE decision to recommend Spinraza
Spinraza will be available on the NHS for everyone!
Spinraza® has been recommended as a standard NHS treatment for all patients with spinal muscular atrophy in England and Wales.
Your amazing #WearYourHurt campaign photos
#WearYourHurt campaign photos. We are an amazing community!
TreatSMA at NICE Conference
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.
We are meeting NICE and NHS today
TreatSMA is talking to NICE and NHS decision makers at a Manchester conference today