[HTML][HTML] The elusive promise of myostatin inhibition for muscular dystrophy
KR Wagner - Current opinion in neurology, 2020 - journals.lww.com
Current opinion in neurology, 2020•journals.lww.com
… 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. … Inhibition of myostatin as a potential therapeutic for
muscular dystrophy was first studied in the mdx mouse, which has a premature stop codon
in the gene for dystrophin and thus is a model of Duchenne muscular dystrophy (DMD) and
Becker muscular dystrophy (BMD) [20] . Mdx mice lacking myostatin had increased myofiber …
trial experience to date of these inhibitors in muscular dystrophy, and the potential reasons
for the lack of observed translation. … Inhibition of myostatin as a potential therapeutic for
muscular dystrophy was first studied in the mdx mouse, which has a premature stop codon
in the gene for dystrophin and thus is a model of Duchenne muscular dystrophy (DMD) and
Becker muscular dystrophy (BMD) [20] . Mdx mice lacking myostatin had increased myofiber …
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.
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