Family with Muscular Dystrophy Heartbroken After Awaiting FDA's Approval on Drug

Photo: The Verdin Family. Source: Newsweek.

What might have been their only option for their children has been rejected by the FDA. This is the story of the Vertin family, and their three children with Duchenne muscular dystrophy.

This fatal genetic disorder has affected many young boys across the country. An example is the case of Betty Vertin’s children, who have Duchenne muscular dystrophy and were waiting for FDA’s final decision on an experimental drug that has helped them improve their muscular strength. Her children, Rowan, Max and Charlie, have participated in a clinical trial for a new treatment that was thought to keep their condition stable for a period of time.

The children, Charlie, Max and Rowan have seen their muscles being slowly affected by the condition since they were born. It is expected that, with this evolution pattern, they will no longer be able to walk in a few year's time.

On September 28th, these three kids went to the Food and Drug Administration building with their mother in order to request the possibility of taking a drug, of which they have taken through a clinical trial that they participated in since they were very young. The truth is that although the kids were already taking the medication that was still under development, they would need an approval from the FDA in order to keep taking it.

If the agency did approve the consumption of the drug, Max, Rowan and Charlie along with many other children with the same condition would have been able to benefit from it. However, the drug was rejected by the FDA in late October. This wasn't the first time this drug has been submitted for an approval, and this is the third time it has been rejected by the government organization. According to the FDA committee, the data about the drug’s effectiveness has been found inconclusive, which is why it won't participate in another clinical trial.

But, this drug has been able to stabilize the Vertins’ condition, which was the main argument presented to the committee when looking for approval back in September. It is also important to mention that the Vertins were not the only family to testify in front of the FDA committee, there were more than a dozen families at the meeting, all looking for the approval to keep taking “Ataluren,” which is the name of the new treatment for Duchenne muscular dystrophy.

The story of the Vertins

In an interview, Betty Vertin, the mother of Max, Rowan and Charlie, said that it all started out very innocent for them. Max was born a big infant, about 10 lbs and nearly 2 ft. long. She recalls that even doctors thought that those “missed milestones” when he was a baby were due to his big body and that he will sooner or later catch up as he grew up. Nevertheless, once he started going to preschool, the difference between him and the rest of the children became more obvious and easy to detect.

Betty remembers that while other kids were jumping and running around, Max was physically behind. It was not until he was 4 years old that he was tested and diagnosed with Duchenne muscular dystrophy. And, at this point in time, Betty was not aware that this disease even existed. Duchenne muscular dystrophy is not a common condition in the United States, nor is it worldwide. In the United States, only about 15 boys out of every 100,000 are diagnosed with Duchenne muscular dystrophy, or its milder form called Becker muscular dystrophy.