Author: Andi Thornton
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 them. Apply for a Free Wizzybug They are also in the process of developing new…
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…
UK SMA Patient Registry Update
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…
Treatsma website reaches national news in Bhutan
Treatsma website reaches national news in Bhutan.
Update on Roche’s SMA global clinical development programme: New global combination study to begin in early 2022
We are pleased to share with you the initiation of MANATEE, a new global Phase 2/3 clinical study that aims to evaluate the safety and efficacy of GYM329 (RO7204239), an investigational anti-myostatin antibody targeting muscle growth in combination with risdiplam, in Spinal Muscular Atrophy (SMA).
Living with SMA – Hitting Our Fourties
TreatSMA Trustee Andi Thornton speaks with SMA UK Trustee Toby Mildon about mental health, treatments, University and work, health care and much more
New trial announced by Biogen
New Clinical Study Planned to Evaluate the Potential Benefit of an Investigational Higher Dose of Nusinersen in People with SMA Previously Treated with Evrysdi® (risdiplam)
New trial announced by Biogen
New Clinical Study Planned to Evaluate the Potential Benefit of an Investigational Higher Dose of Nusinersen in People with SMA Previously Treated with Evrysdi® (risdiplam)