A small study was done that indicated that a drug called Eteplirsen could help treat the symptoms of Duchenne Muscular Dystrophy. A mother has started a petition asking for the FDA to approve the drug. One of her sons was in the study and improved. Her other son was not in the study and cannot have access to the drug.
Sarpeta is a company that makes a drug called Eteplirsen. It was designed to treat the symptoms of Duchenne Muscular Dystrophy. New drugs are not simply distributed to pharmacies and placed on the shelves of drug stores. There are a series of things that must happen first, including one or more studies that test the efficacy of the drug.
A small study was done that included 12 patients. Each patient was a boy who had Duchenne Muscular Dystrophy and who still had the ability to walk. They were split into three groups. Four boys received a placebo. Four boys received a high dose of Eteplirsen. Four boys received a lower dose of the drug.
Researchers measured how far the boys could walk in six minutes. This measurement was taken at the beginning of the trial and again after 48 weeks of treatment. The boys who received Eteplirsen were able to walk, on average, 21 meters farther than the boys who were getting the placebo.
Duchenne Muscular Dystrophy is one of nine different types of Muscular Dystrophy. It is characterized by progressive muscle weakness and wasting. Children who have it experience muscle weakness in the legs and pelvis and lose muscle mass. They also lose the ability to walk and the ability to breathe. There is no cure for this disease.
One of the boys in the study was a 10 year old named Max. His brother, Austin, is 13 years old, and was not included in the study. This is because Max still had the ability to walk when the study started but Austin had already lost that ability.
Their mother, Jen McNary, watched as Max got stronger. He was once dependent on a wheelchair, but no longer needs it, thanks to the Eteplirsen that he is still taking. According to Jen McNary, “He’s no longer riding the handicap bus to school”.
At the same time, she watches her older son, Austin, become weaker. This must be heartbreaking for all of them! Jen McNary has started a petition at Change.org that is titled: “FDA: Please, approve the medicine my boys need to survive – both of my sons deserve to live.”
The hope is that the petition will get enough signatures for it to be sent to the FDA and that they will accelerate the approval process for Eteplirsen. It could mean a world of difference for children who have Duchenne Muscular Dystrophy.
Image by Lucario(F) on Flickr