The attitudes and expectations of patients with SMA are changing with regard to innovative therapies

SMA is currently experiencing a therapeutic revolution, with more and more new molecules on the market or about to be approved. This crippling neuromuscular disease affects several thousand patients in Europe. Many of them are active in European patient associations, most of which are affiliated with SMA-Europe. 

This associative alliance reports, in an article published in February 2021, the results of a survey of 4,749 people concerned. The responses to 1,474 questionnaires from 26 European countries were used and compared with those of a similar survey conducted in 2015, still at the initiative of SMA-Europe. Even if a recruitment bias cannot be ruled out (13% of respondents were included in a trial or clinical study), participation in therapeutic trials in general is a consensus, as is the acceptance of innovative therapies. Respondents believe that the current classification into four sub-types of SMA (from I to IV) is no longer relevant on the one hand and that the simple stabilization of their symptoms still constitutes therapeutic progress on the other hand. The issue of access to treatment, which varies widely from country to country, is also debated. Lastly, many are now waiting for the establishment of bi- or triple therapies specific to SMA, proof that personalized medicine is underway. 


Understanding European patient expectations towards current therapeutic development in spinal muscular atrophy. N Gusset, C Stalens, E Stumpe et al. Neuromuscul Disord. 2021 (Fév).