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?
A couple of weeks ago we had the honour of hosting Dr John Bach for an exclusive webinar looking at how to manage the respiratory system of SMA sufferers.
Roche have released new data which shows that Risdiplam significantly improves motor function after 24 months in people between the ages of 2 and 25 years, this includes both type 2 and type 3 patients. In addition the data also shows that after 12 months of patients previously treated he continues to show a rapid and sustained increase in SMN protein levels.
We have received further clarity statement from the NHS England Clinical Panel for nusinersen with regards to access compassionate program for Risdiplam.