Myotonic dystrophy type 1 (DM1) is a rare, genetic,
progressive muscle disease distinguished by myotonia. People living with
DM1 typically experience progressive weakness of major muscle groups,
which can affect mobility, breathing, heart function, speech, digestion, vision, and cognition. The severity of symptoms and pace of
progression varies. There are currently no disease-modifying therapies
approved to treat DM1.
Each person living with DM1 has a unique and powerful story to tell
Living with DM1 profoundly impacts every part of people’s lives, from work and
socializing to family life. Community members have shared their moving perspectives with our team, including what it would mean to them to have a potentially transformative
therapy for their disease.