Risdiplam is effective in treating babies with SMA type 1
Risdiplam has been proven safe and effective in treating babies with SMA 1
Category: Updates
Pre-approval access programme to risdiplam in the UK
Update on Roche’s pre-approval access programme to risdiplam and how it will be implemented in the UK
Roche makes risdiplam available globally through a pre-approval access programme
Roche is making risdiplam available through a global Pre-Approval Access Programme
Physio with Marion Episode 3
TreatSMA hosted another Physio with Marion session on Sunday, and thanks to Marion and all of those who took part in asking questions, it was another great success which saw questions and views from around the globe, over 1000 by the end of the evening.
Zolgensma to be made available globally under a managed-access programme
Avexis will soon make Zolgensma available for free under a global managed-access programme.
Invitation to take part in Patient Reported Outcome Measures research project
Calling all parents/carers of children under 12, children between the ages of 12-18 and also adults or carers of adults to participate in an ongoing study funded by Biogen.
Unconfirmed reports about Novartis ending branaplam development
TreatSMA is unable to confirm media reports on stopping branaplam development at this stage.