We are so sorry to hear of the continued heightened distress in the SMA Community caused by NICE / NHS England’s decisions and communications to date. We are doing all we can to obtain a reply to the letter sent by clinicians and the patient groups on 14th June. This was sent following consultation, advice and a joint decision that this step was the most promising way to obtain a change to the MAA.
Read full storyToday we have co-signed two letters to address the concerns that are being widely expressed about the nusinersen Managed Access Agreement (MAA).
Read full storyThe FAD will form the guidance to the NHS and the committee papers summarise the information it considered in the process of getting to this stage. We note that the draft MAA still requires some further clarity and development. Jointly with MDUK and TreatSMA we have already raised concerns around this and NICE and NHS England have committed to respond to these at the earliest possible time.
Read full storyThis makes a second ground-breaking treatment for SMA possible in the USA for these children and any who are pre-symptomatic. It is a one-time injection into the blood designed to address the genetic root cause of SMA by replacing the faulty or missing SMN1 gene.
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