UK campaign for access to treatments and support for those with spinal muscular atrophy
The UK SMA Patient Registry has asked TreatSMA to reach out to the community to help the research team at Columbia University, New York who are currently running a project focused on fatigue in SMA. The survey criteria is for eight years old and above and not restricted to any type of SMA or any country.…
Data from pivotal SUNFISH study showed increases in motor function observed during the first year were maintained through the fourth year, while the overall rate of adverse events continued to decrease Data confirm long-term efficacy and safety profile of Evrysdi in a broad range of people with Type 2 and non‑ambulant Type 3 SMA More…
Designability has reached out to TreatSMA to say they have lots of Wizzybugs available and if children are in need of independent mobility from the ages roughly of 14 months to 4/5 years old then please get in contact with them. Apply for a Free Wizzybug They are also in the process of developing new…
Pathfinders Neuromuscular Alliance are offering a free training programme to people with neuromuscular conditions to help them become confident Independent Employers of their own PAs. It is run with support from Skills for Care and ACAS. It is improving hugely popular with people on Direct Payments, and Pathfinders have reserved up to eight places exclusively…
We are delighted to announce changes in the UK SMA Patient Registry! These developments will see the collection of information from adults and parents of children and young people living with SMA about their experience of daily life, their activities and quality of life. Also known as patient-reported outcome measures or PROMs, this information will…
We are delighted to announce changes in the UK SMA Patient Registry! These developments will see the collection of information from adults and parents of children and young people living with SMA about their experience of daily life, their activities and quality of life. Also known as patient-reported outcome measures or PROMs, this information will…
This is a operation setup to help SMA families in Ukraine by SMA Poland. It went live this morning on the 27th February 2022. The link below has been shared with us, it has further details and has been written in Ukrainian. There is now a resource centre setup by SMA Poland for Ukrainian SMA…
We are so pleased to announce that the Scottish Medical Consortium has released its decision to fund Risdiplam for use on the NHS in Scotland to treat 5q SMA.
We are so pleased to announce that NICE, NHS England and Roche have reached agreement on a Managed Access Agreement (MAA) for Evrysdi (Risdiplam) for children and adults with type 1, 2 and 3 SMA. The purpose of the MAA is to ensure access to treatment for the SMA community whilst further data is collected.…
