Brothers with rare terminal muscle disorder fear their future after the FDA denies the drug that keeps them walking

Two brothers suffering the same debilitating disorder face a greater risk of muscle deterioration and early death after the US Food and Drug Administration declined a drug that has kept them walking.

Elliot Johnson, 14, and Henry, 11, suffer from Duchenne muscular dystrophy, a rare form of the disease that causes muscle weakness and leaves more than 90 percent of patients unable to walk by 15 and has an average mortality age of 25.

The FDA refused to approve the protein restoration therapy used to treat their specific condition last month, saying an additional clinical trial would be needed to prove the drug works.

The makers of the drug have filed an appeal but if it fails, the Johnson boys will be off the medication that they’ve taken for almost half their lives as early as December.

The FDA pulled the trial drug for the third time citing the data is inconclusive. This was after the company PTC Therapeutic’s year-long effort at bringing it to the market under the name Translarna.

Mother of the boys, Joanna Johnson, told Daily Mail Online both of her sons have taken the oral medications three times a day since her oldest son was five and the younger boy was seven.

She said the drug, Ataluren, has had the most profound effects on her oldest Elliot, especially when he was younger.

‘He went from not being able to jump to clearing two feet and from spider crawling up the stairs to walking up,’ she said.

The family fears what will happen if the drug is pulled.

Joanna said her sons have asked how long it would take until they can no longer walk.

Duchenne muscular dystrophy (DMD) is a rare genetic mutation categorized by progressive muscle degeneration and weakness.

The disease is mainly in boys and affects one in 3,500 infants worldwide.

It is a severe type of muscular dystrophy and symptoms usually begin between three and five years old and worsen until a patient can no longer walk and function on their own.

More than 90 percent of patients are wheelchair-bound by 15.

Life expectancy is usually in the 20s, though more and more sufferers are living into their early 30s.

Ataluren is the drug designed to treat patients with what is called ‘nonsense mutations’ that prevent the body from producing a key protein needed for muscle development.

‘We’re involved in something now that is making a difference and the thought of starting from scratch is frustrating,’ Joanna said.

Elliot was diagnosed in the summer of 2007 and shortly after, the parents took Henry for a test who was less than a year old at the time.

Once Elliot was diagnosed, the doctors said there was a 50 percent chance that Henry would also have Duchenne muscular dystrophy.

When Elliot turned five, he began the 48-week Ataluren trial and Henry waited until he was seven.

The initial 48-week trial expired and the brothers have been in the extension program for years since.

They travel to the Children’s Hospital of Philadelphia near their home for blood work every eight weeks and take their oral medicine regularly three-times-a-day.

Joanna said she also fears new trials will mean further travel to different hospitals for each son in Ohio and Florida.

The mother-of-two said she has reached out to key players of the FDA via social media to tell her sons’ stories and explain why this drug is making a difference for her and other parents.

Their doctors at CHOP are behind the drug and signed a letter confirming they have seen patients progress and it merits approval.

This clinical trial has been ongoing for more than a decade and Joanna worries it may take up to five years for another trial of this drug to be proven.

She stressed an urgency of time – that if the FDA waits for new trials to be conducted, it may be too late for her boys.