German clinicians have compiled data from the literature in order to assess the efficacy and safety of vamorolone, a new-generation synthetic corticosteroid recently authorised in Europe and the United States to treat Duchenne muscular dystrophy (DMD):
- data from 210 DMD patients treated with vamorolone at a dose of 2 mg/kg/day or 6 mg/kg/day were collected and analysed,
- the product’s efficacy in timed tests (speed of ascent of 4 steps, time taken to rise from the ground) was confirmed, particularly at the highest dose,
- vamorolone appears to have less of a negative impact on statural growth than other steroids, but does not protect against weight gain or insulin resistance.
Despite these encouraging results, the authors emphasise the lack of external validation of the data and of objective data to assess the long-term effect of this new medication.
