We are a UK initiative of people living with spinal muscular atrophy (SMA) and their parents and carers who joined hands to improve the diagnostics, standard of care, social support, and access to treatments in this severe genetic disorder.
We support the efforts to speed up diagnostics and introduce treatment as early as possible. Early introduction of pharmacological therapies offers the highest chance of preventing the disease manifesting itself. We support the introduction of routine newborn screening for SMA as well as wide availability of carrier screening.
Standard of care
TreatSMA aims that hospitals across the country are able to offer the highest standard of medical care to those with SMA. We promote the Standard of Care document and protocols, so as to reduce the risk of severe complications of spinal muscular atrophy and improve the quality of life.
Access to treatments
Everyone with SMA has a right to treatment.
We work to ensure that people with all types of the SMA get the opportunity to access effective treatments. We co-operate with all other stakeholders in the UK SMA community in order to bring about broad access to Spinraza (nusinersen), the first approved treatment for SMA, and all future therapies.
We advocate that those affected by SMA receive an appropriate level of social support, including access to physiotherapy, hydrotherapy, occupational therapy, supportive tools and equipment – everything that reduces the burden of SMA and makes life with this severe disease more bearable.
How we work
We do not believe that sitting back and waiting or hoping for the government to provide us with everything will get it to our children or ourselves.
Our campaigns highlight the devastating effects that spinal muscular atrophy has on those affected by the disease and shows how the treatment and support can improve their quality of life. For many, it is literally the difference between life and death.
Most of all, we encourage people with SMA, their friends and families to work together and fight together. For the first time ever we have hope! Without everyone getting on board our voices might be lost and the effects of that could be devastating!