Campaign for Access to Treatments for Spinal Muscular Atrophy

• 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 than 8,500 people—from newborns to the over 60s—have been treated with Evrysdi, which is now approved in more than 90 countries worldwide

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…

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 for TreatSMA members on the courses, with access code TreatSMA23.

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 be captured in short online questionnaires in the patient registry.

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…

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…

It is becoming more apparent than ever that there is a lack of access to physiotherapists for children and adults in the community that specialise in SMA or have knowledge of and training in the condition.