First in-human study of golodirsen for Duchenne muscular dystrophy shows safety and biologic activity

Histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Cross section of muscle shows extensive replacement of muscle fibers by adipose cells. Credit: Public Domain

This first-in-human study of golodirsen showed its long-term safety and biologic activity in patients with Duchenne Muscular Dystrophy (DMD). The approved exon-skipping therapy is designed to enable the production of functional dystrophin proteins, as described in the peer-reviewed journal Nucleic Acid Therapeutics.

Golodirsen was tested in…

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News Source: https://medicalxpress.com/news/2021-11-in-human-golodirsen-duchenne-muscular-dystrophy.html