NICE and NHS England and NHS Improvement have taken advice from clinical experts and consulted with patient advisory groups (Treat SMA, MDUK and SMA UK) to review the potential impact of COVID-19 on the delivery of the Managed Access Agreement (MAA) and have released the following statement.
A new clinical trial of Spinraza is about to start in the next few months. Codenamed DEVOTE, it will measure the safety and efficacy of Spinraza administered in much higher doses and to a different schedule. Biogen clearly looks for…
Many people reached out through their PAG to contact clinicians, and as a result MDUK has compiled the following regarding muscle wasting conditions. please see below for information written and compiled by MDUK which we would like to share.
TreatSMA hosted another Physio with Marion session on Sunday, and thanks to Marion and all of those who took part in asking questions. This was a special addition which had the usual questions and answers but more…..
Today, European Medicines Agency (EMA) recommended a conditional approval of the breakthrough gene therapy drug Zolgensma® for treating spinal muscular atrophy across the European Union.
An article was recently published in the Lancet providing data from an observational study of adults between the age of 16 and 65 with SMA. This is the first verified data on adults and could prove important in further discussions regarding extending the MAA. Further details below.
Members of our SMA community along with the most vulnerable members in our society which the coronavirus poses the greatest threat to have had enough.
On Sunday 22nd of March, the Prime Minister, Boris Johnson, announced that the UK Government would be setting up local hubs where extremely vulnerable people would be able to register for help with advice, medications and also food supplies.