This year, TreatSMA wanted to present a range of topics for the community, but they were also particularly keen to remember and acknowledge those that fought/fight and got the community where it is today, not just in regards to SMA but disability itself. The ongoing battle for equality and disability rights fought by so many should not be forgotten.
In this session Marion focused on those who have more physical strength and those with type 3. As ever lots of questions and lots of discussion, please do take a look.
A new trial aimed at patients with type 3 SMA is being launched by Catalyst Pharmaceuticals for those living in both Italy and Serbia. The trial is designed to look at the efficacy of a drug called amifampridine.
A new phase 4 trial for those who receiving gene therapy is planned to find out if additional treatment with Spinraza(R) could improve outcomes. Does this move spells that in future a multitreatment approach would be standard?
TreatSMA are really happy to announce Marion Main has formally joined the team and will take on the role as “TreatSMA Consultant Physio Therapist”
c4c (conect4children) is a large collaborative European network that aims to facilitate the development of new drugs and other therapies for the entire paediatric population.
While treatments are obviously important physio is absolutely essential for keeping healthy and benefiting from treatments. Marion this week talks about physio and adults.
We were extremely grateful and privileged to have Lee join us for an interview talking about his experiences receiving Spinraza.