The drug is typically started between ages 4-6 at a dose of 0.75 mg/Kg. However, steroid cause may side effects to Duchenne boys, including weight gain and behavioral problems.
How does it work? We’re not sure, but we think the medication stabilizes muscle membranes and inhibiting cytotoxic T-cells.
Side effects are mediated by binding sites in the cell nucleus which lead to initiation of metabolic pathways :
The new drug VBP-15 is molecularity very similar to Prednisone, but lacks the 11 beta hydroxy arm, which reduces the metabolic side effects, without affecting the beneficial membrane stabilizing effects:
VBP-15 has been shown to improve muscle strength and function in an animal model of DMD without the metabolic side effects of Prednisone:
Clinical trials in humans are expected to begin in 2013-14.
Watch this space for more information.