Author: Andi Thornton
Macedonia will fund Spinraza treatment
The government of Macedonia has agreed to provide Spinraza treatment to all those with spinal muscular atrophy.
Hungary will fund Spinraza treatment
Hungary has agreed to provide Spinraza treatment to all children with SMA.
Thank you to Fairfax Academy
It gives us enormous pleasure, on behalf of everyone in the team here at TreatSMA, to say a massive thank you to the Students and Staff at Fairfax Academy, Birmingham for their generous donation.
Letter to NHS England from the medical and patient communities
Efforts continue on bringing about changes to the NHS commissioning policy on nusinersen. Last night almost 40 clinicians and the four UK SMA groups sent another letter to NHS England. The letter underlined our position that nusinersen treatment under the Expanded Access Programme should be provided to all those who have type 1 SMA.
To Whom It May Concern
I wish you knew the turmoil my heart is in with every single waking beat. I wish you felt the lump in my throat caused by the overwhelming fear of defeat. I wish you saw what I see when I look into my child’s eyes And how beautiful, innocent and pure they are that you…
Community response to NHS letter
Today TreatSMA sent a reply to the NHS letter received last month that defended the draconian funding restrictions for compassionate access to nusinersen in spinal muscular atrophy (SMA).
Preparing for action
Dear Community, After we all partook in sharing the TreatSMA video to raise awareness of SMA, we have been flooded with many questions about how can the community help. The fact is: NHS will not pay this much money for any drug voluntarily. We have between 1–2 thousand SMA-ers in the UK. Which means that…
RG7916 increases SMN protein 2.5-fold
PTC Therapeutics informed that the experimental drug RG7916 increased the SMN protein 2.5-fold in SMA type 2 and 3 patients who took part in the SUNFISH clinical trial.