How Do Dissociated Steroids Work?

Dissociated steroids, also known as dissociative steroids, are types of glucocorticoid medications used in the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years and older. Currently, the first-in-class dissociated steroid is vamorolone (brand Agamree), approved by the FDA in October 2023, and expected to become available in the market in the first quarter of 2024.

Duchenne muscular dystrophy is a severe, genetic, progressive muscle wasting disease that can be fatal. DMD is caused by the complete absence of dystrophin, a protein that is vital for the integrity of muscle cells, due to mutations in the dystrophin gene. Dissociated steroids reduce disease-related inflammation in DMD.

Glucocorticoids are potent anti-inflammatory agents used as first-line treatment for DMD, however, they produce many undesirable side effects. Dissociated steroids have been developed to retain the anti-inflammatory and immunosuppressive efficacy of steroids, and dissociate these activities from their detrimental side effects.

Dissociated steroids are used for the treatment of Duchenne muscular dystrophy in patients 2 years of age and older. The only dissociated steroid currently approved by the FDA currently is vamorolone, an oral suspension.

Side effects of dissociated steroids include the following:

• Vomiting
• Cough
• Headache
• Development of Cushing syndrome and cushingoid features such as:
• Moon face
• Abnormal fat deposits
• Weight gain around the midsection and upper back
• High blood sugar (hyperglycemia)
• Adrenal insufficiency
• Vitamin D deficiency
• Psychiatric disorders such as:
  • Agitation
  • Aggression
  • Irritability
  • Abnormal behavior
  • Anxiety
  • Altered mood
  • Sleep disorder
• Repetitive actions (stereotypy)
• Nasal inflammation (rhinitis)
• Diarrhea
• Increased appetite
• Increased risk of infection
• Increase in blood pressure
• Electrolyte disturbances
• Decrease in bone mineral density
• Eye infections
• Cataracts
• Elevated intraocular pressure
• Glaucoma, a condition that damages the optic nerve
• Stunting of growth and development in children
• Increased risk of blood clot blocks (thromboembolism)
• Kaposi sarcoma, a type of cancer
• Severe allergic reactions (anaphylaxis), rare

Information contained herein is not intended to cover all possible side effects, precautions, warnings, drug interactions, allergic reactions, or adverse effects. Check with your doctor or pharmacist to make sure these drugs do not cause any harm when you take them along with other medicines. Never stop taking your medication and never change your dose or frequency without consulting your doctor.

Generic and brand names of dissociated steroids include:

Agamree

vamorolone