SMA Daily Life Study

SMA Daily

SMA Daily Life Study

The daily lives of people living with SMA are impacted by much more than motor function. Factors like fatigue, pain, and mental health affect the well-being of those living with SMA and may be valuable outcomes measures when assessing treatment.

The results of the SMA Daily Life Study will help us better understand how these factors relate to each other and how they impact individuals’ daily lives. 

The Daily Life Study supports the current shift towards research on “patient-relevant outcomes”, stressing the importance of establishing how individuals living with SMA define well-being and quality of life in their own terms. Our goal is to develop tools to measure these and other outcomes that may be relevant to those living with SMA. This will guide our advocacy efforts for equal access to optimal treatment and care in Europe.

 

Scroll to the end of this page for details, participation instructions, and FAQs.

 

 

 

 

"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

At a glance

  • The study opens in March 2023 for English speakers all over Europe.  

  • Participants need to be 16 years of age or older and own a smartphone. 

  • The study will include approximately 150 participants. 

  • Participants respond to questionnaires through a smartphone application, multiple times a day for several days. This allows us to see how patient-reported outcomes change overtime and influence daily life. 

  • The study looks at emotions, bodily feelings, and situational factors to understand when and why they happen and how they influence daily life. 

  • This study is carried-out in collaboration with Tilburg University in the Netherlands and supported by the Cure SMA Industry Collaboration.

Detail

Why do we need the SMA Daily Life Study?

  • Assessments of treatment success 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. This study will provide insight into how these factors impact individual daily lives by assessing people’s well-being intensively, over a short period of time.  

  • The impact of factors like fatigue, pain and mental health varies greatly not only between different individuals, but also within the same person. These measures may also change for the same person at different times. The Daily Life Study will capture these changes and analyse how different factors relate to each other. (For instance, how does experiencing pain influence how tired you are feeling?) 

  • The results from this study will help us to better understand what patient-relevant outcomes look like in the daily lives of people living with SMA. 

  • With this information, we can develop tools to measure patient-relevant outcomes in a reliable and valid way. With these tools, we will advocate for patient-relevant outcomes to be included in SMA care and research.

This study has been approved by Tilburg University's Ethics Review Board and complies with the European General Data Protection Regulation (GDPR) (EU) 2016/679.