Select a Country or Region

DISEASE

STABILISATION

Without intervention to stabilise the disease, people living with type II/III spinal muscular atrophy (SMA) will see a progression of their disease over time.1,2 Stabilising SMA is important to stabilise and maintain motor function for as long as possible.

The characters shown are real patients and the required consent to use their stories has been obtained from the patients and families. Photographs are for illustrative purposes only.

DISEASE STABILISATION REPRESENT MAJOR PROGRESS

People living with SMA, have become aware that disease stabilization is key to maintaining an optimal quality of life.1

According to a study published in 2012, people living with SMA will continue to experience disease progression, if they continue to lack the suitable care and intervention.2

It has become clear to both people living with SMA and their caregivers that it is vital to stabilize and maintain motor function for as long as possible, to allow people with SMA to stay independent and live life on their own terms.2

HERE IS WHAT PEOPLE LIVING WITH SMA
AND THEIR CAREGIVERS THINK...

A recent survey of people affected by type II/III SMA described the impact on their quality of life and their expectations relating to care options1

822

PARTICIPANTS

52% (425) of the represented people living with SMA were Adults aged 20 and above

436

PEOPLE LIVING
WITH SMA

383

PARENTS/
CAREGIVER

3

UNDISCLOSED

PRESERVING AND MAINTAINING ABILITIES FOR PATIENTS WITH SMA

The perspectives and experiences of people living with SMA are critical to the development of new treatments. In a recent survey of people living with type II/III SMA and their caregivers it was shown that:1

felt that if there was a new treatment available that could stabilise their disease course, it would represent a major improvement to their lives.1

The
TOP 3

abilities patients chose to STABILISE were:

The
TOP 3

abilities patients chose to IMPROVE were:

THEIR PERSONAL EXPERIENCE WITH SMA DICTATES WHAT THEY WANT OUT OF CARE

See what they found important:1

RESPIRATORY FUNCTION

IMPROVE PROXIMAL
MOBILITY/ FUNCTIONALITY

MUSCLE STRENGTH

Improvement or stabilisation, including coughing and swallowing

Getting up, balancing, walking, jumping, running, climbing stairs

Stabilisation or improvement

RESPIRATORY FUNCTION

Improvement or stabilisation, including coughing and swallowing

IMPROVE PROXIMAL
MOBILITY/ FUNCTIONALITY

Getting up, balancing, walking, jumping, running, climbing stairs

MUSCLE STRENGTH

Stabilisation or improvement

References

1. Rouault F, et al. Disease impact on general well-being and therapeutic expectations of European Type II and Type III spinal muscular atrophy patients. Neuromuscul Disord 2017;27(5):428-438.

2. Kaufmann P, et al. Prospective cohort study of spinal muscular atrophy types 2 and 3. Neurol 2012;79:1889-1897.