Ariana Boswell

All articles by Ariana Boswell

• What genetic changes cause DMD?

  The DMD gene is the largest known gene, making it more prone to mutations.

• What are mdx mice, and why are they used in research?

  Mdx mice have a mutation in the gene that codes for the protein dystrophin, which is mutated in patients with DMD.

• More awareness needed of uncommon severe comorbidities in DMD

  A recent study identified many severe comorbidities in patients with DMD, including gastrointestinal, cardiovascular and kidney conditions.

• Eteplirsen delays cardiac decline in DMD clinical trials

  A recent study found that individuals with DMD who receive eteplirsen have slower declines in cardiac function than those who do not.

• World Duchenne Awareness Day 2025: How to get involved

  World Duchenne Awareness Day takes place on September 7 each year to raise awareness of DMD and advocate for improved research and education.

• Study shows givinostat is effective in patients with DMD long-term

  An open-label extension of the EPIDYS clinical trial found that givinostat is safe and delays disease progression in patients with DMD.

• Case report: Patient with DMD receives successful heart transplant

  A young man with DMD received a heart transplant at 18 years old and reports positive outcomes after 3.5 years.

• Small molecule drug improves muscle health in DMD mice

  A recent study found that inhibiting ADAMTS-5, a cartilage-degrading enzyme, with GLPG1972 supports muscle function in a mouse model of DMD.

• Sarepta shares plans to improve safety of Elevidys

  Following two fatal cases of acute liver failure associated with ELEVIDYS, Sarepta is taking steps to improve the safety of the drug.

• REGENXBIO shares positive findings from DMD gene therapy trial

  Preliminary results from the AFFINITY DUCHENNE trial show that the gene therapy RGX-202 improves functional performance in patients with DMD.