UNLOCK THE POTENTIAL BENEFITS OF SPINRAZA
The SPINRAZA clinical study program is the largest in spinal muscular atrophy (SMA) to date
The effectiveness of SPINRAZA was established in the pivotal ENDEAR study and supported by multiple clinical studies. Results were measured differently based on factors such as the age and physical ability of individuals in the clinical studies.
For individuals with infantile-onset (Type 1) and presymptomatic SMA in the ENDEAR and NURTURE studies, results included the achievement of developmental motor milestones.
The CS2/CS12 studies focused on individuals with later-onset (Types 2 and 3) SMA, evaluating improvements and maintenance of motor function over time, as well as specific motor skills—for example, walking and upper limb function.
Click through the tabs below for a closer look at the study results across a range of
individuals with SMA.
What are controlled and open-label studies?
Clinical studies are designed to determine whether a medication is effective and to monitor for any side effects. In a controlled study, there are treated and untreated groups, and the researchers and patients do not know who is receiving the drug. In an open-label study, both researchers and patients know what treatment is being given.