241 patients enrolled on EAMS
We received news yesterday that 241 people are now enrolled on the EAMS for Risdiplam.
New and innovative assistive technology launched to help those who struggle with using computers
We don’t normally do blog posts of this nature, but we felt that this particular piece of assistive technology is so good that we wanted to share it with the community.
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.
Sibs: Camp in the Cloud for siblings and their families
A SMA community member has sent us the following information, they thought it would be useful for the SMA community.
Zolgensma Q&A
We have received further information from NHSE on Zolgensma. We will continue to update you all as we receive information. Please follow link below for the Q&A. Zolgensma Q and As
NHSE announce Zolgensma treatment centres
We are please to share communication from NHSE about the progress regarding access to Zolgensma. The selection of the four centres which will be administering the treatment has been completed.