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

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

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

Columnist Shalom Lim reviews "Supervision," a play that delves into questions about privacy, dependency, and dignity.

The determination comes after recruitment and dosing in certain trials were paused after a young patient died due to acute ...

Columnist Patrick Moeschen, anticipating his own historic anniversary, celebrates champions of the disability rights movement ...

Lisa Littleton, who is the mother of two sons with Duchenne muscular dystrophy, one passed and one living, is a full-time caregiver. She describes the difficult balance between pushing for care and ...