The core team at TreatSMA, met on Saturday 13th July to discuss ongoing activities into accessing treatment for SMA and also to discuss our future plans.

Treatsma Meeting 13 July 2019

TreatSMA met in Peterborough on Saturday to review status of activities with treatments for SMA. We wanted to provide you a quick update on what was discussed and various outcomes. Please see below.

Research update:

The team reviewed on ongoing development of treatments for SMA and an update will be posted shortly. MAA included patients: As a significant result of activities over the past two years approx 75% patients are now eligible for the treatment with Spinraza.

There remain concerns around the speed in which this treatment will be made available in hospitals due to the processes and procedures that need to be put in place to administer Spinraza. These processes are not just the actual administration, but under the MAA hospitals are required to record efficacy, and therefore infrastructure around the administration also needs to be made available.

While we know a few centres are investigating ways to introduce the treatment for children and adults, we know that other centres may take a little longer. While the drug itself has been funded by NHS England the infrastructure around administration still needs to be agreed on a hospital by hospital basis. TreatSMA will aim to identify those centres which are having difficulties introducing treatment and will use our network and knowledge to come up with solutions and ways to get treatment started.

Please alert us if you are aware of the difficulties to access if elegible.

Non-MAA:

TreatSMA continues to work with Biogen, NICE, NHSE, clinical community and other charities to expand access to Spinraza or alternative treatments. The current campaign to work with MPs on the provision of Spinraza to patients not included in the MAA is continuing. TreatSMA are pulling together a database of patients identified as non-eligible and identifying clinical experts open to give a second opinion. We will continue to investigate processes, campaign and other feasible routes to seek review of the current decision.

TreatSMA Adult Group:

TreatSMA will be working with SMA adults to form a group focused on adult needs for care and treatment. More news on this very soon.

This was viewed by the team as an exciting meeting and with the many treatments advancing there are more opportunities to ensure every SMA individual will have access to a treatment. We remain resolute in our position of ensuring all people receive treatment.