Table 1.

Immunomodulators in DMD. NFAT, nuclear factor of activated T cells; PDE, phosphodiesterase.

Drug/compound	Target	Pathological process	Preclinical trials	Clinical trials/use
*Current treatments*
Prednisone, deflazacort	NF-κB, others	Anti-inflammatory	Yes	Yes
VBP15	NF-κB, membrane protection	Anti-inflammatory, sarcolemma stability	Yes	Yes*
Cyclosporine	NFAT	Anti-inflammatory	Yes	Yes^†
Azathioprine	Purine synthesis	Anti-inflammatory	Yes	Yes^†
Poloxamer	Membrane protection	Sarcolemma stability	Yes	Yes^‡
Gene therapy	Dystrophin replacement	Sarcolemma stability	Yes	Yes
Exon skipping	Dystrophin replacement	Sarcolemma stability	Yes	Yes
TLR7/8/9 antagonists	TLR7/8/9	Anti-inflammatory	Yes	No
NEMO peptide	NF-κB	Anti-inflammatory	Yes	No
Infliximab	TNF-α	Anti-inflammatory	Yes	No
IL-2/anti–IL-2 complex	T_regs	Anti-inflammatory	Yes	No
Pentoxifylline	PDE inhibitor	Anti-fibrotic	Yes	Yes
Pirfenidone	TGF-β signaling	Anti-fibrotic	Yes	No
Losartan	Angiotensin type 1 receptor inhibitor	Anti-fibrotic	Yes	Yes
Lisinopril	Angiotensin-converting enzyme inhibitor	Anti-fibrotic	Yes	Yes
Anti–IL-6	IL-6	Anti-inflammatory	Yes	No
Anti-myostatin antibodies	Myostatin	Anti-fibrotic, hypertrophy	Yes	Yes
Cromolyn	Mast cells	Membrane stability	Yes	No
*Future options*
Chloroquine	Lysosomal pH	Anti-inflammatory	No	No
Eculizumab	Complement C5	Anti-inflammatory	No	No
Rapamycin	T_regs +Akt/mTOR	Anti-inflammatory, regeneration	Yes	No
Plerixafor	CXCR4	Anti-inflammatory	No	No
IL-10	Alternatively activated macrophages	Anti-inflammatory	No	No
Anti-osteopontin antibodies	Osteopontin	Anti-inflammatory, anti-fibrotic	No	No
Candesartan	Angiotensin type 2 receptor inhibitor	Anti-fibrotic	No	No

*Phase 1, 2015. †Not effective. ‡Effective in heart.