Delivery 7–10 daysAgamree (vamorolone) oral suspension
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Description
💊 Prescription dissociative steroid for Duchenne muscular dystrophy in patients from 2 years of age.
▪︎ Vamorolone oral suspension 40 mg/mL, 100 mL bottle with dosing syringes and a bottle adapter
▪︎ Manufacturer: Catalyst Pharmaceuticals
▪︎ FDA approval: October 2023
🔬 How it works: glucocorticoids remain the backbone of treatment because they damp the inflammation accompanying muscle fibre breakdown and extend the ability to walk by years — but they arrive as one package, bringing growth delay, fragile bones, weight gain and cataract with them. Vamorolone binds the same glucocorticoid receptor without activating that entire set of programmes: the anti-inflammatory action is preserved while the pathways leading to growth delay and bone loss are engaged less. It also blocks the mineralocorticoid receptor, which is linked to fluid retention. 📊 Evidence: VISION-DMD (JAMA Neurol 2022) in 121 boys, mean age 5.4 years — time-to-stand velocity 0.05 m/s versus −0.01 m/s on placebo (p=0.002); growth percentile fell by 1.88 on prednisone and rose by 3.86 on vamorolone (p=0.02), with bone turnover markers declining on prednisone but not on vamorolone. The 48-week extension (Neurology 2024) maintained the motor effect, and children switched from prednisone recovered growth.
⚠️ Prescription-only, prescribed by a neurologist. It replaces a conventional glucocorticoid rather than adding to one. Adrenal suppression occurred in every trial group including vamorolone, so the drug is never stopped abruptly and doses are adjusted during illness or surgery; body mass index also rises. Superiority over prednisone on motor outcomes was not tested.
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