Long-term outcomes for females with early-onset dystrophinopathy.
Saskia L S Houwen-van OpstalRamon O TakMaaike PelsmaFrederik M A van den HeuvelHermine A van DuyvenvoordeEdith H C CupLilian T L SieJohan S H VlesImelda J M de GrootNicol C VoermansMichel A A P WillemsenPublished in: Developmental medicine and child neurology (2022)
Females with early-onset dystrophinopathy experience a wide range of impairments, comorbidities, limitations in activities, and restrictions in social participation. The whole spectrum should be acknowledged in the healthcare setting. Neuromuscular and cardiac follow-up are indispensable. Additional respiratory assessment and rehabilitation care are expected to improve health status and support daily activities and participation.