We have come a long way from treating the symptoms of SMA to being able to stabilise or improve motor function in children with pre-symptomatic or symptomatic SMA.1 SPINRAZA® helps pre-symptomatic children achieve milestones unexpected in Type I or II SMA and more consistent with normal development.1 The greatest beneﬁt is observed in those with earlier treatment initiation.1
Treatment with SPINRAZA® is also associated with a signiﬁcant improvement in overall survival in children with Type I SMA compared to untreated patients.1
4 years*Pre-symptomatic SMA
Treated with SPINRAZA®
*Age at time of photoshoot
The characters shown are real patients and
the required consent to use their pictures/
stories has been obtained from the patients
and families. Photographs are for
illustrative purpose only.
Individual results may vary based on several
factors, including severity of disease,
initiation of treatment, and duration of
Dr. Liesbeth de Waele, discusses the new possibilities for treatment of SMA, including in pre-symptomatic and symptomatic children with SMA Type I.
1. SPINRAZA® Summary of Product Characteristics. January 2020.