Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.*
*Pivotal trials did not include adult patients with spinal muscular atrophy (SMA).
In a SPINRAZA® (nusinersen) pivotal trial, people with later-onset SMA showed meaningful improvements in overall motor function as measured by Hammersmith Functional Motor Scale—Expanded (HFMSE) compared to placebo at 15 months. Additionally, in a pivotal trial in people with
early-onset SMA, a majority of infants were motor milestone responders at 13 months.
Learn more about clinical and real-world trial data
Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
The safety profile of SPINRAZA
has more than 8 years of
follow-up data that is consistent with
those previously reported.
SPINRAZA is delivered directly to the
central nervous system (CNS) where
motor neuron loss begins. After 4
initial loading doses, SPINRAZA is
given 3 times a year.
have been treated with
there’s someone from almost every
age group who has taken
†Based on commercial patients, early access patients, and clinical trial participants through May 2022.
‡SPINRAZA pivotal studies included patients from 3 days to 16 years of age at first dose and did not include sufficient numbers of subjects aged 65 and older to determine whether they respond differently from younger patients.