TreatSMA featured on charitable.radio
Charitable.radio is an amazing station focused on raising awareness of charities from all walks of life. You can listen on the Internet or using your smart device by simply asking it to play charitable radio.
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.
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.