Author: Andi Thornton
What a community! Thank you.
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).
Physio with Marion – Equipment
Today Marion looked at equipment, how it’s used and what you should be looking for in general.
EAMS update and how you can help
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…
EAMS update and how you can help
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…
Survey to explore the experiences of people who are on Risdiplam.
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.
Biogen Release a Community Update
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
Survey on Access to Treatment for Spinal Muscular Atrophy
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.
Roche presents new 2-year data for Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)
Roche presents new 2-year data for Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)