Update on AveXis’s global Managed Access Programme for Zolgensma
The Zolgensma access programme cannot currently be extended to the UK
Category: Updates
TreatSMA Needs You
We are very humbled and proud to be nominated for the Community Organisation Award at the annual National Diversity Awards 2020. We have been nominated by you, the community, who stood beside us, and fought for what we believed in, for what we all believed in. Without your drive and your spirit, we would not…
TreatSMA Rare Disease Day Event 2020 – Watch Now
TreatSMA hosted its own event celebrating Rare Disease Day on February 29. There are estimated 8000 rare diseases across the world affecting over 300 million people, so we decided to showcase some other conditions in addition to SMA.
TreatSMA Rare Disease Day event EXCLUSIVE – This Saturday, 29th February, don’t miss it!
As we published earlier in the week TreatSMA is proud to be supporting the Royal Holloway’s 10th Annual Rare Disease Day Event on Wednesday 26th in support of Rare Disease Day. However, the excitement doesn’t stop there.
Physio with Marion Episode 5
TreatSMA hosted another Physio with Marion session on Sunday, and thanks to Marion and all of those who took part in asking questions, his week’s session focused on standing and walking and there were lots of great questions.
TreatSMA sponsor Rare Disease Day event – 26 February 2020
TreatSMA are proud to announce that we will be one of the sponsors for Royal Holloway’s 10th Annual Rare Disease Day Event on Wednesday 26th of February.
Roche’s risdiplam showed significant improvement in motor function in people aged 2-25 with type 2 or 3 spinal muscular atrophy
Roche released part 2 of the SUNFISH data which shows significant improvement both type 2 and type 3 patients. Read on for further details.
Physio with Marion Episode 4
TreatSMA hosted another Physio with Marion session on Sunday, and thanks to Marion and all of those who took part in asking questions, it was another great success which saw questions and views from around the globe.