Duchenne muscular dystrophy (DMD) is a genetic neuromuscular disease that primarily affects young males. Patients with DMD are unable to produce dystrophin, a crucial protein found in myocytes, leading to a loss of muscle support and integrity. Corticosteroids are the standard supportive treatment for DMD; however, there is a high demand to expand the number of safe, effective pharmacologic options. Recently a surge of new therapeutics for DMD is offering hope to patients. A variety of these new medications, such as stop codon readthrough agents, exon-skipping agents, and utrophin modulators, aim to replace dystrophin in myocytes. Other new therapeutics aim to prevent or repair muscle damage caused by the absence of dystrophin. This review provides an update on the medications being investigated in DMD.

Traumatic brain injury results in significant morbidity and mortality, and there is an urgent need for neuroprotective medications that can prevent the persisting symptoms and disabilities following injury. Several existing pharmacotherapies have been targeted for off-label benefit in traumatic brain injury, as these agents are well characterized and commercially available, easing the process of clinical trial development. Despite promising results in animal models, clinical trials have demonstrated minimal benefit. One possible reason for these failed translations could be that drug selection, characterization and dosing are not routinely established in the appropriate early phase trials before larger scale testing. Examining how recent trials may have bypassed these steps may help future trials to more definitively determine the efficacy of potential therapeutics.