You will be aware on the 8th August we remember all those who we lost to SMA. Our angels who we continue to remember with love. As a type 1 father who lost my son Toby, it is not just one day, I remember him everyday.
We are overjoyed to be able to bring you the news that the FDA has approved risdiplam for the treatment of types 1, 2 and 3 with no age restrictions.
On Friday evening TreatSMA hosted a webinar with Michelle Woods who is a SENDCo at a primary school in Kent. She has been a primary school teacher for 20 years and a SENDCo for 6 years. Michelle currently works in…
Another amazing session with Marion, this episode focusing on pain.
This year, TreatSMA wanted to present a range of topics for the community, but they were also particularly keen to remember and acknowledge those that fought/fight and got the community where it is today, not just in regards to SMA but disability itself. The ongoing battle for equality and disability rights fought by so many should not be forgotten.
In this session Marion focused on those who have more physical strength and those with type 3. As ever lots of questions and lots of discussion, please do take a look.
A new trial aimed at patients with type 3 SMA is being launched by Catalyst Pharmaceuticals for those living in both Italy and Serbia. The trial is designed to look at the efficacy of a drug called amifampridine.
A new phase 4 trial for those who receiving gene therapy is planned to find out if additional treatment with Spinraza(R) could improve outcomes. Does this move spells that in future a multitreatment approach would be standard?