What the trial was testing
The trial enrolled 25 patients with spinal muscular atrophy. The study was sponsored by Biogen and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was initial testing (phase 2). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
Babies on the higher dose hit motor milestones never seen without treatment.
The Lancet · 2016 · NCT02386553
These findings — that led to motor milestone gains and improved survival vs. natural history in infant SMA — were published in the The Lancet and represent the headline result of the study.
Researchers tracked outcomes across 25 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with spinal muscular atrophy, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Nusinersen (Spinraza) is FDA-approved and available now for SMA in children and adults, given as a spinal injection every four months after loading doses. Two other treatments — onasemnogene abeparvovec (Zolgensma) and risdiplam (Evrysdi) — are also FDA-approved. Ask a pediatric neurologist about which fits your child best.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open spinal muscular atrophy trials
HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness
This single-arm pilot study will assess the preliminary effectiveness of an intensive motor skill intervention (HABIT-ILE) combined with functional strength training (FST) in children with SMA who are receiving disease-modifying therapies. Participants will attend a HABIT-ILE + FST summer camp for 6 hours per day over a 3-week period, totaling 90 hours of training.
ECoG BMI for Motor and Speech Control
Test the feasibility of using electrocorticography (ECoG) signals to control complex devices for motor and speech control in adults severely affected by neurological disorders.