Blog posts
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.
The powerpoint slide Marion used in the latest episode of Physio with Marion titled Choosing a private physiotherapist for you or your child (In the UK) is now available as a PDF document.
Treatsma website reaches national news in Bhutan.
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).