Open access
Oct 2026
Blood biomarkers identified with multi-omics combined with clinical and muscle imaging in oculopharyngeal muscular dystrophy
Oculopharyngeal muscular dystrophy (OPMD) is an adult-onset, slowly progressive muscular dystrophy characterized by dysphagia, eyelid ptosis, and proximal limb weakness. Clinical trial readiness in OPMD requires development of sensitive indicators of disease progression and potential response to therapies in de...
I. Smith, Matthew Triolo, Karim Karimi et al.
· Orphanet Journal of Rare Dis... · 0 citations