Ariana Boswell

All articles by Ariana Boswell

• (Z)-Endoxifen receives Rare Pediatric Disease designation from FDA

  (Z)-Endoxifen, a drug being developed by Atossa Therapeutics for DMD, recently received Rare Pediatric Disease Designation from the FDA.

• Deramiocel trial yields positive results in patients with DMD

  Findings from the HOPE-3 trial indicate that deramiocel may improve cardiac and skeletal muscle health in patients with DMD.

• Obestatin alleviates symptoms and improves muscle health in DMD models

  In mouse and human cell models of DMD, treatment with obestatin repaired muscle damage and improved cellular function.

• Can adults with DMD live independently?

  For individuals with DMD, “living on your own” can take on many different meanings.

• Regular use of lung volume recruitment therapy low in DMD

  Fewer than half of participants with DMD who were prescribed LVR therapy adhered to their treatment protocol, a study found.

• Trehalose may improve effects of microdystrophin therapy in DMD

  Researchers found that trehalose further improved cellular function in animal models of DMD treated with microdystrophin therapy.

• Synthetic molecule prevents DMD in mouse model

  A study found that a synthetic compound known as a BB polymer may protect skeletal and cardiac muscles from damage in DMD.

• Experimental drug improves gut-muscle crosstalk in DMD mice

  Researchers found that targeting gut microbiome dysfunction in a mouse model of DMD improves muscle health and function.

• How do genetic variants impact DMD treatment and prognosis?

  The type of genetic variant a person with DMD has can impact which therapies they are eligible for.

• What genetic changes cause DMD?

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