How many clinical trials are tracked for Duchenne muscular dystrophy?

57 trials are tracked, 10 with published results.

Duchenne muscular dystrophy Research

Duchenne muscular dystrophy: what the trials found

Novel genetic and pharmacological interventions show varying efficacy in functional outcomes. Ataluren demonstrated a statistically significant improvement in the 6-Minute Walk Distance (6MWD) at week 72 when analyzed using an Intent-to-Treat population, though results were not significant in the modified Intention-to-treat group ¹. In another trial, Ataluren did not show a significant change in 6MWD at week 48 ⁴. Givinostat showed a statistically significant improvement in the 4 Standard Stairs (4SC) climb after 18 months of treatment, but did not significantly improve Time to Rise From Floor or 6MWT scores ⁷.

Gene-based therapies and protein expression studies show measurable biological activity. Delandistrogene moxeparvovec resulted in a statistically significant increase in dystrophin protein expression at week 12 ⁵ and an 81.18% change in Dystrophin Positive Fibers (PDPF) by day 90 ¹⁹. While delandistrogene moxeparvovec showed a statistically significant improvement in Time to Rise From the Floor at week 52, it did not significantly improve North Star Ambulatory Assessment (NSAA) scores ⁵.

Supportive and secondary interventions include respiratory and immunological management. Lung Volume Recruitment (LVR) was associated with improvements in health-related quality of life over 2 years ². Subcutaneous seasonal flu vaccines demonstrated statistically significant geometric mean titer ratios across various strains ³. Prednisone treatment did not result in statistically significant changes in Rise From the Floor velocity, NSAA scores, or patient satisfaction ⁶.

Other investigated agents include Edasalonexent ¹⁰, Viltolarsen [12, 18], RO7239361 (which showed no significant change in NSAA scores at week 48) ¹¹, and Eplerenone, which did not significantly impact left ventricular strain ⁸.

For the clinician treating this condition

Ataluren showed statistically significant improvements in 6MWD in specific populations, but results were inconsistent across different trial cohorts.
Delandistrogene moxeparvovec successfully increased dystrophin protein expression and improved Time to Rise From the Floor, though it did not significantly impact NSAA scores.
Givinostat demonstrated significant improvement in 4 Standard Stairs (4SC) climb but no significant effect on 6MWT or other mobility metrics.
Flu vaccinations showed statistically significant titer ratios, supporting their role in immune management.

AI synthesis of 11 cited trials, updated Jun 23, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.