Author: Andi Thornton
Unsatisfactory response from NHS England
TreatSMA has received a formal response from NHS England to the letter sent by the UK medical community about the Expanded Access Programme of nusinersen.
SMA Awareness Day
Today 30/9 the world celebrates SMA Awareness Day. Do you know what SMA stands for?
A new trial of gene therapy in SMA announced
Today Avexis announced the start of a new clinical trial of gene therapy in SMA.
Spinraza will be offered in Italy to all with SMA
Yesterday the government in Rome decided that Spinraza will be offered in Italy to all those with spinal muscular atrophy.
Important update on branaplam development programme
Another exciting update: branaplam, an experimental molecule for SMA from Novartis, is finally moving ahead.
First open TreatSMA meeting
On Moday 18 September TreatSMA families held a meetup in order to get updates about treatments and discuss actions needed for fast and broad access to SMA therapy in the UK.
RG7916 moves to pivotal clinical trial in SMA types 2 and 3
Exciting developments! Roche’s new experimental drug for SMA is moving to late-phase clinical trial in SMA types 2 and 3.
Our SMA Story
Vinnie was born by C section on the 24/03/2015 and he was absolutely perfect in every way but I could never shake that feeling that he was too perfect. Always in the back of my head I had this horrible that something was terribly wrong. By the time he was 4 weeks old I was…