Avidity's delpacibart etedesiran (del-desiran) was granted orphan drug status in Japan as a treatment for myotonic dystrophy type 1 (DM1).

Learning life skills to handle Duchenne muscular dystrophy can include health management, working with caregivers, and ...

Health Canada granted priority review to an application seeking approval of vamorolone, marketed in the U.S. as Agamree, for ...

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