Unmet Therapeutic Needs of Non-Ambulatory Patients with Duchenne Muscular Dystrophy: A Mixed-Method Analysis.

Anne L R SchusterNorah L CrossnohereRyan FischerPatricia FurlongJohn F P Bridges

Published in: Therapeutic innovation & regulatory science (2022)

Non-ambulatory Duchenne patients want new treatments that improve upper limb functioning and body system functioning, and not exclusively regaining ambulation. The PUL-PROM can be used as a patient-centric measure that accounts for the needs of later-stage Duchenne patients.

Keyphrases

end stage renal disease
duchenne muscular dystrophy
ejection fraction
newly diagnosed
chronic kidney disease
blood pressure
upper limb
peritoneal dialysis
prognostic factors
muscular dystrophy
case report
patient reported