. Author manuscript; available in PMC: 2019 Oct 1.

Published in final edited form as: Matrix Biol. 2017 Nov 27;71-72:174–187. doi: 10.1016/j.matbio.2017.11.009

Table 3.

Drug treatments directed at sequelae of muscle damage evaluated in mouse models

Inhibition target	Mouse	Treatment	Observed Bene- fits	Comments	Ref.
Inflammation	dy^W/dy^W	Prednisolone (10 µg/gm/wk p.o.)	Survival increase from 66 to 90% at 24 weeks; no change in weight; 16% increase in grip strength.	Long-term steroid use many side-effects that may make this drug problematic in LAMA2-MD.	[104, 111]
Protein degradation (proteosome genes)	dy^3K/dy^3K & dy^2J/dy^2J	Bortezomib (0.4 mg/kg i.v.)	Median survival, weight and fiber size in dy^3K/dy^3K mildly increased with decreased regeneration.	No beneficial effect in dy^2J/dy^2J mouse.	[105, 106]
Apoptosis	dy^W/dy^W & dy^2J/dy^2J	Omigapil (0.1–1 mg/kg oral)	Improves respiration, locomotion and weight and protects from early mortality Modest reduction of fibrosis.	Under phase 1 clinical trial for LAMA2 and collagen-VI deficiencies.	[99, 100]
Fibrosis	dy^W/dy^W & dy^2J/dy^2J	Losartan (0.6 mg/ml drinking water)	Decreases TGF-β signaling. Reduced fibrosis and inflammation. Improved locomotion, strength and weight.	Losartan is an angiotensin II receptor type 1 antagonist shown to block pro-fibrotic action of TGF-β.	[101–103]
Other	dy^W/dy^W	Doxycycline (6 mg/ml drinking water)	Median survival increased from 32 to 70 days; small weight increase; improved behavior and histology.	Tetracycline derivatives may benefit the dystrophy by apoptosis inhibition.	[112]