28 trials tracked for spinal muscular atrophy: 9 in phase 3 or 4 and 6 with published results. The most-cited published study has 1433 citations.
Showing the 28 most-cited and recently-updated of 28 trials. Browse the full registry →
Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.
Nusinersen has demonstrated significant improvements in motor function across several trials. In infants with SMA, it was associated with a statistically significant increase in CHOP-INTEND scores compared to sham controls 5, and high rates of achieving motor milestones according to HINE and WHO criteria 10. Clinical assessments also showed significant increases in HFMSE scores 3 and RULM scores 6. Additionally, nusinersen was associated with improved motor milestones and function as measured by CHOP-INTEND 21.
Onasemnogene abeparvovec-xioi showed clinical efficacy in specific cohorts, including a significant number of participants achieving the ability to stand alone for at least 3 seconds 4 and high rates of event-free survival at 14 months of age 4. Risdiplam was associated with significant improvements in infant motor function, with high percentages of infants achieving scores of 40 or higher on the CHOP-INTEND scale and meeting specific sitting milestones 16.
OAV101 was evaluated in multiple trials; while it showed measurable changes in HFMSE and RULM scores at week 52 8, other assessments focused on vital signs such as systolic and diastolic blood pressure 7. Additionally, a flu vaccine was shown to produce significant geometric mean titer ratios across various strains 2.
AI synthesis of 13 cited trials, updated Jun 21, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.
