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ARTICLE | Distillery Therapeutics

Musculoskeletal

October 18, 2018 6:45 PM UTC

Patient sample, cell culture and mouse studies suggest inhibiting CB1 could help treat Duchenne muscular dystrophy (DMD). In quadriceps tissue samples from pediatric DMD patients, CB1 expression levels were higher than in samples from age-matched healthy volunteers. In patient-derived myoblasts, the CB1 antagonist rimonabant increased expression of myogenesis markers including troponin T type 1 (skeletal slow; TNNT1; ANM) compared with vehicle. In a mouse model of DMD, rimonabant decreased myofiber loss in gastrocnemius muscle and increased motor coordination, skeletal muscle strength and myofiber regeneration in gastrocnemius muscle. Next steps could include testing additional CB1 antagonists in mouse models of DMD.

In 2007, Sanofi withdrew Acomplia rimonabant from the market for obesity due to psychiatric side effects...

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