Campaign for Access to Treatments for Spinal Muscular Atrophy

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…

Today TreatSMA sent a reply to the NHS letter received last month that defended the draconian funding restrictions for compassionate access to nusinersen.

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…

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.

TreatSMA has received a formal response from NHS England to the letter sent by the UK medical community about the Expanded Access Programme of nusinersen.

Today Avexis announced the start of a new clinical trial of gene therapy in SMA.

Yesterday the government in Rome decided that Spinraza will be offered in Italy to all those with spinal muscular atrophy.