Duchenne muscular dystrophy is a very serious and fatal disease that slowly destroys every muscle in the body. This rare disease affects one out of every 3,500 people and mostly affects young boys who will normally be completely reliant on a wheelchair by the young age of 12. Most people with this condition only live until age 25. Read more about this story here.
Fortunately a new drug, called Exondys 51, has allowed one teenage boy in the US to continue to miraculously walk at age 16. Billy is one of twelve young boys in the US who has been participating in a clinical trial for six years for this drug.
The Exondys 51 drug was created by two professors in Western Australia: Sue Fletcher and Steven Wilton. Wilton was the one who first came up with the idea to create a drug that can trick a person’s cells into skipping over the Duchenne muscular dystrophy mutation.
It took twenty-five years, but finally the drug became a reality. Even though the scientists who made the drug were from Australia, the drug itself has not been approved in there. However, the drug was approved quickly in the US by the FDA in 2017. The two scientists are now working hard to get the drug approved for commercial sale in their home country.
The chief executive of Muscular Dystrophy of Western Australia, Hayley Lethlean, has spoken out and said that there are 100 people in Western Australia alone with the disease and about 800 in Australia as a whole.
The drug has had issues being approved in Australia because of limitations with commercialization, funding, patent issues, and lack of environment for clinical trials. The biggest hope is to have the drug available. Families, patients, and caretakers in Australia with Duchenne muscular dystrophy hope that seeing Billy with such a successful outcome will speed up the approval process and offer hope for those still waiting for the drug.
“It feels good to be walking for this long and I’m glad I can,” Billy says.
Billy graduates in 2 years and his mother cannot wait to see him walk across the stage accept his diploma.
