Experimental drug KER-065 aims to slow muscle decline in duchenne muscular dystrophy

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Diagnosis of DMD, defined as the presence of phenotypic features at screening consistent with DMD AND documented mutation in the dystrophin gene consistent with the diagnosis of DMD using a clinically validated genetic test. * Receiving a stable regimen of systemic CS (including, but not limited to, prednisone, prednisolone, deflazacort, or vamorolone) for at least 90 days before screening. * Body weight of ≥ 25.0 kg. Ambulatory Participants Only (Cohort A1 and A2): * Ambulatory, defined as able to walk independently without assistive devices. * Able to TTR in \< 10 seconds. * Has a NSAA score ≥ 15 points. * Cohort 2 only: Documentation of a stable dose of an approved exon-skipping therapy. Nonambulatory Participants Only (Cohort N1): * Nonambulatory, characterized as being unable to ambulate for a minimum of 3 months before first dose with onset of nonambulatory status AND a NSAA walk score of 0 and inability to perform the 10MWR. * PUL v2.0 entry item score of 3 to 5, inclusive. Key Exclusion Criteria: * Clinical symptoms or signs of cardiomyopathy or heart failure. * Exposure to any approved or investigational dystrophin restoration gene therapy product. * Exposure to any approved or investigational dystrophin restoration product other than gene therapy (Except for exon-skipping therapy for Cohort A2). * Exposure to any approved or investigational histone deacetylase inhibitor, antimyostatin therapy, therapy targeting transforming growth factor-beta ligands, or cell-based therapy. * Use of any other pharmacological treatment, except for CS * Treatment with immunosuppressant therapy (other than CS) * History of fracture of the upper limb Nonambulatory Participants Only (Cohort N1): * Elbow-flexion contractures \> 30° in both upper extremities. * Forced vital capacity (FVC) of \< 50% or requirement for daytime or nocturnal ventilation, except for nocturnal non-invasive ventilation AND inability to perform consistent FVC measurements within ± 15% during paired testing.