PTC Therapeutics informed that the experimental drug RG7916 increased the SMN protein 2.5-fold in SMA type 2 and 3 patients who took part in the SUNFISH clinical trial.
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.
Today 30/9 the world celebrates SMA Awareness Day. Do you know what SMA stands for?
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.