Today we learn that treatment for other rare diseases were approved for cystic fibrosis in Scotland and Batten disease. Thoughts and congratulations go out to everyone affected by or working hard to receive treatment for these diseases from TreatSMA.
Acaster Lloyd Consulting Ltd, an independent research consultancy specialising in patient research, on behalf of a pharmaceutical company, is conducting an online survey for adults and caregivers of children with a medical diagnosis of spinal muscular atrophy (SMA) to understand views about the importance of different aspects of treatment for SMA and need your help.
Now that the MAA has been released attention turns towards monitoring progress with regards to the implementation of treatment for those currently eligible. Eligible patients should have access to treatment within 90 days of the NICE guidance being released subject to any clinical or commissioning issues. This is what we know so far.
Recently, acting as part of TreatSMA, Gary McKie (trustee) asked the community if anyone had a panthera chair that a young North East family could loan, to keep their sons arm strength up, as his consultant had recommended sourcing a manual chair.
Biogen UK has released new community statement with regards to access to Spinraza in the UK. TreatSMA, SMAUK and MDUK have asked important questions and this statement provides some insight in the process and what to expect.
In celebration of the approval of Spinraza the Annabelle Rose Foundation organised a party called the Cirque du Spinraza where many of the community attended on Saturday.
TreatSMA visited Roche yesterday to talk about Risdiplam and how we can support them through the approval and appraisal process.
Hansard official record of the House of Commons debate on Spinal Muscular Atrophy has now been issued.