SMA Daily Life Study

SMA Daily

SMA Daily Life Studies

Fatigue, pain and mental health are factors impacting the well-being of individuals living with SMA and are considered as “patient-relevant outcomes”.

They are getting more and more research attention and might play an important role as valuable outcome measures when assessing treatments. This study will support the shift in research towards more “patient-relevant outcome measures” and consequently, our advocacy efforts for equal access to optimal treatment and care in Europe.

 

 

 

 

"SMA and SMA treatments impact many aspects of the daily lives of people living with SMA. In order to fully understand this impact, we must study it in people’s actual daily lives, together with all the complexities and chaos that life entails."

Dr. Yasemin Erbas, Vice-President SMA Europe

In focus

Why do we need the SMA Daily Life Study?

  • Assessments of treatment successes should be driven by what is important for people living with a disease
  • In SMA, such patient-relevant factors are, among others, fatigue, pain and mental health 
  • Following people living with SMA over time (i.e., in a longitudinal study) will give insights how these factors are impacting the daily life of individuals.
  • Research from fields outside of SMA show that these factors (i.e., constructs) have a high level of between-person, but also within-person variability. Moreover, at the within-person level, they may have temporal dependencies with other variable constructs. To unravel this, research focusing in detail on individuals is necessary.
  • The results from this study will help us to better understand how patient-relevant outcomes are manifested in the daily lives of patients
  • This can help with the development of tools that will allow for measuring them in a reliable and valid way, taking into account both between-person differences and within-person changes.

Detail

What is the Study?

  • The study will be launched in 2022
  • The study will include approximately 150 participants from five European countries. 
  • In this study, we are using the Experience Sampling Method (ESM). ESM is a research method through which participants are prompted multiple times a day for several consecutive days to respond to an electronic questionnaire through a smartphone application. 
  • The SMA Daily Life Study will include psychological, social and contextual variables by repeatedly assessing these factors to unravel their predictors and consequences
  • This study is carried-out in collaboration with Tilburg University in the Netherlands and supported by the Cure SMA Industry Collaboration.