Join Joshua In His Fight For The Life Changing Drug Viltepso

Product Description

Perth, Western Australia, Australia

Supporters

Joshua is 8 years old and suffers from Duchenne Muscular Dystrophy. Duchenne Muscular Dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact.

Children with Duchenne have difficulty jumping, running, and walking. Other symptoms include enlargement of the calves, a waddling gait, and lumbar lordosis (an inward curve of the spine). Later on, the heart and respiratory muscles are affected as well. Progressive weakness and scoliosis result in impaired pulmonary function, which can eventually cause acute respiratory failure. Currently there are NO SURVIVORS of DMD.

In August 2020, Viltepso, an “exon skipping” drug that targets a section of DNA called exon 53 was approved by the FDA for treatment of individuals who have a confirmed mutation of the DMD gene that is amenable to a therapeutic strategy called exon 53 skipping. The drug has also been approved for use in Japan.

Although treatment with Viltepso will not cure DMD, it could slow progression of the condition, which, in turn, could extend the length of time individuals with DMD could walk, eat independently, and breathe without assistance.

Approved in the US. Approved in Japan. Not approved in Australia. We can rush COVID vaccines through the Therapeutic Goods Association (TGA) approval process but not this one??

We have tried the Health Minister, the TGA and have even contacted the pharmaceutical company in Japan and America. We are constantly hitting road blocks have no choice but to take the steps to import the drug ourselves.

An infustion of this drug costs approximately $12,000 a week. We are currently fundraising to secure 6 months supply of this life changing drug for Joshua from Japan. He needs to start taking this drug NOW to give him a chance of beating this terrible condition. DMD is a ticking time bomb – with every day that passes Joshua’s body becomes weaker as his failure to produce dystrophin affects every muscle in his body – even his heart!

Any assistance would be greatly appreciated. If you are unable to donate, please share with your friends and family so we can get this drug for Joshua

For more information on Duchenne Muscualar Dystrophy please visit Save our Sons Duchenne Foundation, the peak body for those living with Duchenne and Becker muscular dystrophy (around 1,000 young people) across Australia.

https://saveoursons.org.au/

Please like Joshua’s Facebook page Join Joshua to follow his journey.

https://www.facebook.com/JoinJoshua4DMD/

Emily Mcleod

10 months ago

$20

Emily Mcleod

10 months ago

$20

Emily Mcleod

10 months ago

$20

Stavros Norman

2 years ago

$50

Peta Kapor

2 years ago

$25

Anonymous

2 years ago

$50

Elleke Bosworth

2 years ago

$50

Rob Inness

2 years ago

$50

Anonymous

2 years ago

$50

Anonymous

2 years ago

$25

Malcolm Charles

2 years ago

$50

Janet Jacqueline Duarte

2 years ago

$25

Anonymous

2 years ago

$89

Wayne Smart

2 years ago

$25

June Pauna

2 years ago

$100

Anonymous

2 years ago

$100

Helen Howard

2 years ago

$25

Isabel Noakes

2 years ago

$50

Karina Hincksman

2 years ago

$50

Kirstie Burrows

3 years ago

$30

Anonymous

Best of luck with your fundraising!!

4 years ago

Peter McBurney

$645

William Jones

$300

Kristy Mcglew

$105

Andrew Stuht

$100

Jo Kettle

$50

Eliot Jones