New long-term data suggests givinostat slows progression in DMD
The latest results from a study suggests that givinostat, a treatment for Duchenne muscular dystrophy (DMD), may help slow down the loss of movement. The study looked at boys with DMD who were taking givinostat along with standard treatment (corticosteroids). Researchers compared their progress with what usually happens in people with DMD who don’t take givinostat.
This type of comparison is called a “natural history study”. It uses existing data to show how the condition normally changes over time without the new treatment. This method is often used in long-term studies of serious conditions like DMD, because it wouldn’t be fair to ask people to take a dummy drug (called a placebo) for many years.
The study followed 194 boys who took givinostat for about one and a half years on average. Some had been on the treatment for more than eight years.
The results showed that givinostat delayed the loss of walking ability by about two to three years. It also helped delay other signs of muscle weakness, like getting up from the floor or climbing stairs. This means that while the condition still gets worse over time, givinostat may slow it down and allow people to stay mobile for longer.
Although these results are encouraging, natural history studies have some limits. They’re not as strong as clinical trials where people are randomly given either the treatment or a placebo. That’s because the groups might not be exactly the same, which makes it harder to be sure the treatment caused the improvements. Even so, this study adds to the evidence that givinostat may help slow down DMD.