The elusive promise of myostatin inhibition for muscular dystrophy.

Abstract:

PURPOSE OF REVIEW:Recent terminations of clinical trials of myostatin inhibitors in muscular dystrophy have raised questions about the predictiveness of mouse models for this therapeutic strategy. RECENT FINDINGS:A variety of myostatin inhibitors have been developed for preclinical and clinical studies. These inhibitors have ameliorated the phenotype of many but not all mouse models of muscular dystrophy. However, randomized double-blinded placebo controlled trials in both pediatric and adult muscular dystrophies have, as of yet, not demonstrated functional improvement. SUMMARY:The present article will review the preclinical promise of myostatin inhibitors, the clinical trial experience to date of these inhibitors in muscular dystrophy, and the potential reasons for the lack of observed translation.

journal_name

Curr Opin Neurol

authors

Wagner KR

doi

10.1097/WCO.0000000000000853

subject

Has Abstract

pub_date

2020-10-01 00:00:00

pages

621-628

issue

5

eissn

1350-7540

issn

1473-6551

journal_volume

33

pub_type

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