Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in bmx model mice

Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in bmx model mice
  Personal take on this article: The article discusses the role of chronic inflammation in worsening Duchenne Muscular Dystrophy (DMD), a condition where muscle weakness occurs due to the lack of a protein called dystrophin. Despite treatments that try to restore dystrophin, inflammation continues, highlighting the need for research models that can help understand and treat this persistent inflammation. The study focuses on certain microRNAs (miRNAs), named inflamma-miRs, that contribute …
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The X-linked Becker muscular dystrophy (bmx) mouse models Becker muscular dystrophy via deletion of

The X-linked Becker muscular dystrophy (bmx) mouse models Becker muscular dystrophy via deletion of
Personal take on this article: Becker muscular dystrophy (BMD) is a genetic disease that affects muscles, caused by mutations in the dystrophin gene. It's not as severe as Duchenne muscular dystrophy (DMD), but there aren't many treatments available for BMD. This is partly because there's no good mouse model for studying the disease and testing potential treatments before trying them on people. To address this, researchers created a mouse model …
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Interrogation of Dystrophin and Dystroglycan Complex Protein Turnover After Exon Skipping Therapy

Interrogation of Dystrophin and Dystroglycan Complex Protein Turnover After Exon Skipping Therapy
Personal take on this article:   Researchers studied the turnover of dystrophin and dystroglycan complex (DGC) proteins in mice with Duchenne Muscular Dystrophy (DMD) after receiving exon skipping therapy. Exon skipping therapies, such as Eteplirsen, Golodirsen, Viltolarsen, and Casimersen, have received accelerated approvals from the FDA for DMD. However, these therapies have shown variable and often insufficient levels of restored dystrophin protein in patients, limiting their effectiveness. To understand the …
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