We joined a session with Roche earlier this week to ask them questions following their recent announcement about risdiplam. The following FAQs have been checked for accuracy with Roche where they relate specifically to risdiplam.
Questions and answers apply to people living in England, Scotland, Northern Ireland and Wales.
Today TreatSMA made submissions to NICE to help with the appraisal of Risdiplam to be made available through NHS. We have included extensive data collected from community (over 300 responses in total, so massive thank you).
NICE have announced they are going to review the MAA Guidance for Spinraza in regards to Type 3 patients.
Today Marion looked at equipment, how it's used and what you should be looking for in general.
You may remember some time ago we posted that MHRA had approved an EAMS process for accessing risdiplam. Just to remind everybody what an EAMS process is, it stands for Early Access to Medicines Scheme, and is designed to provide those who currently have unmet clinical needs to get access to life-saving treatment before it is licensed in the UK.
TreatSMA is asking for help from those that are receiving Risdiplam. We desperately need you to fill in this questionnaire on behalf of yourself or your child to help us in our fight to access Risdiplam here in the UK.
This is a community update from Biogen on the efficacy of Spinraza in patients with SMA Type 2 and 3. Positive improvement were seen in both Types. For more details please see below. Biogen Community Update
SMA Community, TreatSMA needs you! In October we make our Patient Advocate submission as part of the NICE appraisal for Risdiplam. Therefore we need to continue to collect data in order to strengthen our fight.