TABLE 2.
Status of the current therapeutic strategies acting on downstream pathological mechanisms of Duchenne muscular dystrophy (https://www.clinicaltrials.gov).
| Therapeutic strategies | Mechanism of action | Chemistry | Drug route | Current stage |
| Anti-fibrotic | ||||
| Pamrevlumab | Monoclonal anti-CTGF antibody | Antibody | Intravenous | Phase II |
| Losartan | Angiotensin II type 1 receptor blocker | Small molecule | Oral | Phase II |
| Halofuginone | Inhibitor of collagen a1 and MMP2 | Small molecule | Oral | Phase I/II (Suspended) |
| Infliximab | Antibody to human TNF-α | Antibody | − | Pre-clinical |
| Suramin | Inhibits TGF-β | Small molecule | − | Pre-clinical |
| Imatinib Mesylate (Gleevec) | Inhibits TGF-β | Small molecule | − | Pre-clinical |
| Anti-inflammatory | ||||
| Corticosteroids (Prednisone, Prednisolone, and Deflazacort) | NF-κB inhibition | Small molecule | Oral | Phase III |
| Edasalonexent | NF-kB inhibition | Small molecule | Oral | Phase III |
| Vamorolone (VBP-15) | NF-kB inhibition | Small molecule | Oral | Phase II |
| Increlex (R) | Recombinant IGF-1 | Fusion protein | Subcutaneous | Phase II |
| TAS-205 | Hematopoietic prostaglandin D synthase inhibitor | Small molecule | Oral | Phase II a |
| Flavocoxid | NF-kB inhibition | Small molecule | Oral | Phase I |
| Givinostat | Histone deacetylase (HDAC) inhibitor | Small molecule | Oral | Phase I/II |
| Tamoxifen | Estrogen receptor modulator | Small molecule | Oral | Phase III |
| Reduction of muscle damage | ||||
| Ca2+ dysregulation | ||||
| Rimeporide | Sodium–hydrogen exchanger 1 inhibitor | Small molecule | Oral | Phase Ib |
| AT-300 | Blocks mechanosensitive Ca2+ channels | Small molecule | − | Pre-clinical |
| Recombinant Mitsugumin 53 | Facilitates membrane repair at sites of injury | Recombinant proteins | − | Pre-clinical |
| BGP-15 | Hsp72 inducer | Small molecule | − | Pre-clinical |
| Streptomycin | Nonspecific Ca2+ channel blocker | Small molecule | − | Pre-clinical |
| Oxidative stress | ||||
| Coenzyme Q10 | Electron acceptor for NADH and succinate dehydrogenase | Small molecule | Oral | Phase III |
| Idebenone | Antioxidant | Small molecule | Oral | Phase III |
| N-acetylcysteine | Endogenous antioxidant | Small molecule | − | Pre-clinical |
| Muscle ischemia | ||||
| Sildenafil | PDE5 inhibitor | Small molecule | Oral | Phase I/II |
| L-Arginine | Metabolic support | Recombinant proteins | Oral | Phase I |
| Tadalafil | PDE5 inhibitor | Small molecule | Oral | Phase III (Terminated) |
| Muscle atrophy | ||||
| GLPG0492 | β2-Agonist, increases cAMP | Small molecule | Oral | Phase I |
| Urocortin | Increases cAMP | Ca2+-independent phospholipase A2 type β | − | Pre-clinical |
| rAAV1.CMV.huFollistatin344 | Delivery of follistatin using adeno-associated virus | AAV1-Follistatin | Intramuscular | Phase I/II |
| Bone homeostasis | ||||
| Zoledronic acid | Inhibits bone resorption | Small molecule | Intravenous | Phase III |
| Alendronate (ALN) | Improves bone mineral density | Small molecule | − | Pre-clinical |