• A prospective and retrospective analysis of the effects of eteplirsen has been conducted over a total period of 7 years, via 3 successive clinical trials among 12 participants with DMD; the third trial involved 10 of these participants and was conducted in a real-life setting with routine clinical follow up.
• The results showed:
- an extra 2 years of maintained ability to walk, compared to boys with DMD not treated with eteplirsen, taken from patient registries or other trials; the mean age for loss of ability to walk among treated participants was around 15 years;
- a less significant decline in respiratory function with forced vital capacity twice as large among treated participants compared to that of control participants.
• This study confirms the results of eteplirsen at 2 years with respect to walking and breathing obtained in the phase III PROMOVI trial among 78 boys with DMD, published in June 2021.
Voir aussi « DMD : les effets positifs à long terme de l’eteplirsen se confirment »
