Author: Marni Smyth
SMA UK put out a community survey
SMA UK has put out a community survey asking for people’s views on what services and activities they should focus on in the future. You can respond to their survey here https://www.surveymonkey.co.uk/r/SMAUKnext
Lianne joins the team
TreatSMA are extremely happy to announce that Lianne Harris has joined the team as a volunteer to assist with Fundraising and Events Co-ordination.
TreatSMA are now listed on AmazonSmile
We are now listed as a charity on Amazon Smile. By choosing us as your selected charity Amazon will donate 0.5% of the net purchase price (excluding VAT, returns and shipping fees) of eligible purchases to us. You can set us as your chosen charity by clicking here or by going to Settings > AmazonSmile…
Survey for Changing Places International
Ross Hovey (a friend of TreatSMA) is the co-founder of Changing Places International whose aim is to set a global standard for fully accessible toilets.
Novartis has made the decision to discontinue development of branaplam, an investigational oral, once-weekly RNA splicing modulator, for the treatment of spinal muscular atrophy (SMA). This was a difficult decision that was made as the result of rapid advancements in the SMA treatment landscape in recent years.
241 patients enrolled on EAMS
We received news yesterday that 241 people are now enrolled on the EAMS for Risdiplam.
Petition to get Newborn Screening funded immediately for Spinal Muscular Atrophy
A petition has been set up to get Newborn Screening funded immediately for Spinal Muscular Atrophy.
NICE Release Statement on Risdiplam
Earlier today NICE released a statement regarding Risdiplam. NICE’s initial response is to say ‘no’ to funding Risdiplam.