Interrogation of Dystrophin and Dystroglycan Complex Protein Turnover After Exon Skipping Therapy
Sign Up to like & getrecommendations! Published in 2021 at "Journal of Neuromuscular Diseases"
DOI: 10.3233/jnd-210696
Abstract: Recently, the Food and Drug Administration granted accelerated approvals for four exon skipping therapies –Eteplirsen, Golodirsen, Viltolarsen, and Casimersen –for Duchenne Muscular Dystrophy (DMD). However, these treatments have only demonstrated variable and largely sub-therapeutic levels… read more here.
Keywords: turnover; muscle; dystrophin protein; restored dystrophin ... See more keywords
A Proof of Principle Proteomic Study Detects Dystrophin in Human Plasma: Implications in DMD Diagnosis and Clinical Monitoring
Sign Up to like & getrecommendations! Published in 2023 at "International Journal of Molecular Sciences"
DOI: 10.3390/ijms24065215
Abstract: Duchenne muscular dystrophy (DMD) is a rare neuromuscular disease caused by pathogenic variations in the DMD gene. There is a need for robust DMD biomarkers for diagnostic screening and to aid therapy monitoring. Creatine kinase,… read more here.
Keywords: human plasma; clinical monitoring; plasma; monitoring ... See more keywords