Table 3.

Principal currently ongoing alternative approaches to treat DMD

Drug	Description/Activity	Effects	Limitations	Clinical trial	References
Ataluren (PTC124) PTC Therapeutics	Small chemical compound that induces ribosomal read‐through of premature stop codons	Restoration of expression of full‐length dystrophin	Use limited to patients with nonsense mutations (nmDMD)	Phase III completed Conditional approval in Europe	[63, 64]
Givinostat Italfarmaco	Inhibitor of HDAC (enzymes that prevent gene activity), which are constitutively active in DMD muscles	Reduction of necrosis and fibrotic and adipose tissue deposition	No restoration of dystrophin expression	Phase III ongoing	[54, 55, 73]
Idebenone (Catena/Raxone) Santhera Pharmaceuticals	Chemical short‐chain benzoquinone; potent antioxidant and lipid peroxidation inhibitor at mitochondrial level	Expected cardioprotection and improvement of muscle performance and respiratory functions	No restoration of dystrophin expression	Phase III ongoing	[59, 60, 61]
Tadalafil and Sildenafil Eli Lilly and Company	PDE5 inhibitor induces vasodilatation through cGMP signalling activation	Expected improvement of muscle blood flow during physical exercise	No restoration of dystrophin expression Little evidence of benefits	Phase III completed	[56, 57, 76]
Vamorolone (VBP15) ReveraGen BioPharma	Glucocorticoid‐like oral drug with anti‐inflammatory and membrane‐stabilizing properties	Reduction of muscle inflammation No glucocorticoid‐associated side effects	No restoration of dystrophin expression	Phase II ongoing	[72]

cGMP, cyclic guanosine monophosphate; HDAC, histone deacetylase; nmDMD, nonsense mutation Duchenne muscular dystrophy; PDE5, phosphodiesterase‐5.