ZOLGENSMA UPDATE
The pause in access to treatment with Zolgensma in children with type 1 SMA aged older than 12 completed months has been lifted.
Information about Apitegromab – The new treatment from Scholar rock
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.
New SMA Drug in phase 3 trials fast tracked
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).
Do you know about the Council for Disabled Children?
TreatSMA is signposting families to the Legal Handbook 3rd edition uploaded by the Council for Disabled Children.
UK SMA Patient Registry need your help
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…
New four year data for Roche’s Evrysdi reinforce long-term efficacy and safety profile
- 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 Need Our Help
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…
Disabled people learn how to take charge of their own care
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.