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.
There is now a MAA Panel which has been set up for the SMA community in England.
A special edition of Physio With Marion which focused on children’s physio. Marion took lots of questions both from children and parents within the community, definitely worth a watch. Why not make it a family event?