What the trial was testing
The trial enrolled 3 patients with spinal muscular atrophy. The study was sponsored by Marco Capogrosso and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
Researchers followed patients through treatment and into recovery, tracking the outcomes that mattered most for the disease being studied.
What the results showed
Up to 180% strength gains in three adults with SMA from spinal cord stimulation.
Nature Medicine · 2025 · NCT05430113
These findings — that in three adults with SMA after 4 weeks of spinal cord stimulation — were published in the Nature Medicine and represent the headline result of the study.
Researchers tracked outcomes across 3 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
Spinal cord stimulation for SMA is still in development and not yet FDA-approved for this purpose. Three FDA-approved SMA medicines (nusinersen, onasemnogene abeparvovec, risdiplam) are available now. Ask a neurologist about approved options and whether you might qualify for a stimulation trial.
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
Vasodilator and Exercise Study for DMD (VASO-REx)
Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve cardiovascular health) and tadalafil, an FDA-approved vasodilator (to optimize blood flow and muscle perfusion which is impaired and often overlooked in DMD). Target: improved muscle function, vascular health, and DMD treatment.
Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
This is an observational multicenter retrospective and prospective study on natural history of SMA also considering the 'new natural history' secondary to the availability of commercially available therapies. All the patients enrolled to date in the Italian registry, if not part of clinical trials, will be included in the present study.