Updates

The pause in access to treatment with Zolgensma in children with type 1 SMA aged older than 12 completed months has been lifted.

Recently the FDA designated Apitegromb for fast tracking as a treatment for  SMA. We wanted to provide some additional information about this treatment, read on for more information and trial details.

The US Food and Drug Administration (FDA) has granted Fast Track designation for Biohaven’s new anti-myostatin adnectin, taldefgrobep alfa, to treat spinal muscular atrophy (SMA).

TreatSMA is signposting families to the Legal Handbook 3rd edition uploaded by the Council for Disabled Children.

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…

• 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.