Working with Joshua to fight for the life-changing drug Viltepso

Joshua is 8 years old and has Duchenne muscular dystrophy.

Duchenne muscular dystrophy (DMD) is an inherited disorder characterized by progressive muscle degeneration and muscle weakness caused by changes in a protein called dystrophin, which helps keep muscle cells intact.

Children with Duchenne have difficulty jumping, running and walking.

Other symptoms include enlarged legs, a faltering gait and lumbar lordosis (the inward curvature of the spine).

Later, the heart and respiratory muscles are also affected.

Progressive weakness and scoliosis can lead to impaired lung function, which can eventually lead to acute respiratory failure.

At present, there are no survivors of DMD.

In August 2020, the US Food and Drug Administration (FDA) approved exon skip for Viltepso, a DNA fragment that targets an "exon", to treat individuals with a proven mutation in the DMD gene that complies with a treatment strategy called exon 53 skip.

The drug has also been approved for use in Japan.

Although treatment with Viltepso does not cure DMD, it can slow the progression of the disease, thus extending the time that people with DMD can walk, eat and breathe unaided.

Approved in the United States.

Approved in Japan.

Not approved in Australia.

Can we snap up the Covid vaccine through the Therapeutic Drugs Association (TGA) approval process, but not through that process?

We have tried the Health Secretary TGA and have even contacted pharmaceutical companies in Japan and the United States.

We continue to encounter obstacles and have no choice but to take steps to import drugs ourselves.

Injections of the drug cost about $6,000 a week.

We are currently raising funds to ensure that Joshua is provided with this life-changing drug from Japan for six months.

He needs to start taking this drug now to give him a chance to beat this terrible disease.

DMD is a ticking time bomb -- as Joshua fails to produce dystrophin, affecting every muscle in his body -- even his heart, weakening every day.

Any help would be greatly appreciated.

If you are unable to donate, please share with your friends and family so we can get this medicine for Joshua

For more information on Duchenne muscular dystrophy, visit the Save Our Sons Duchenne Foundation, which is the main body for people with Duchenne and Becker muscular dystrophy (approximately 1,000 young people) across Australia.