Novel steroid for treatment of Duchenne Muscular Dystrophy

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The FDA recently approved the use of Vamorolone, a novel corticosteroid, for the treatment of Duchenne muscular dystrophy in patients aged 2 years and above.
The drug enhances steroidal anti-inflammatory action and is a powerful antagonist against mineralocorticoid receptors. It is a safer and more effective alternative to glucocorticoids as it exhibits fewer side effects, such as weight gain, cushingoid appearance, growth retardation, and bone morbidities.

Mechanism of action:

Glucocorticoids have numerous mechanisms of action that are responsible for both positive and negative effects.
Vamorolone is a dissociative steroid that only retains pathways that promote efficacy and does not activate pathways responsible for detrimental side effects.

Dosage and administration:

Dosage: 6 mg/kg once daily, patients weighing more than 50 kg may receive up to 300 mg daily.
Route of administration: directly dispensed into the mouth via a syringe.

Precautions for long-term use:

  • Alterations in endocrine function
  • Immunosuppression and increased risk of infection
  • Alterations in cardiovascular/renal function
  • Behavioral and mood disturbances.



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