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.
We received news yesterday that 241 people are now enrolled on the EAMS for Risdiplam.
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.
A petition has been set up to get Newborn Screening funded immediately for Spinal Muscular Atrophy.
Earlier today NICE released a statement regarding Risdiplam. NICE’s initial response is to say ‘no’ to funding Risdiplam.
A SMA community member has sent us the following information, they thought it would be useful for the SMA community.
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