Today Avexis announced the start of a new clinical trial of gene therapy in SMA.
Yesterday the government in Rome decided that Spinraza will be offered in Italy to all those with spinal muscular atrophy.
Another exciting update: branaplam, an experimental molecule for SMA from Novartis, is finally moving ahead.
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.
Exciting developments! Roche’s new experimental drug for SMA is moving to late-phase clinical trial in SMA types 2 and 3.
Come and join our meetup on Monday 18 September to talk about access to SMA treatments and what we all need to do to receive it.
On 23 August 2017, TreatSMA along with SMA Trust, SMA Support UK and MD UK signed a letter addressed to the Head of Specialised Commissioning Team at NHS England. We urged a review of the published commissioning policy for inclusion of SMA 1 patients in the Expanded Access Programme of nusinersen.
Roche shared an update on the safety of RG7916.