Adem Dani has spinal muscular atrophy but his parents are hoping for a 'life-changing’ drug to be fast-tracked onto the PBS

Adem Dani (centre) with his brothers Zain and Ben.
Adem Dani (centre) with his brothers Zain and Ben.

The almost constant cheeky smile on the face of Adem Dani hides a painful truth.

The five-year-old has spinal muscular atrophy (SMA) and will be confined to a wheelchair for the rest of his life and gradually lose all muscle control.

Adem was diagnosed with the rare inherited genetic muscle wasting disease that is characterised by a loss of nerve cells called motor neurons, at just 14-months-old.

But his mother Shendita Dani had a feeling all was not well when her second-born son kept on “flopping” every time she put him on his bum.

“At first I just thought Adem was a really relaxed baby. He was always smiling and never cried or whinged. I thought I had been blessed,” Mrs Dani said.

“I still feel blessed to have him in my life but it soon became apparent that there was something wrong. We were devastated when the doctor told us Adem had SMA.”

SUPPORT NEEDED: Adem Dani, 5 has spinal muscular atrophy. His family are encouraging people to sign a petition to get the "life-changing" Spinraza drug onto the Pharmaceutical Benefits Scheme (PBS).

SUPPORT NEEDED: Adem Dani, 5 has spinal muscular atrophy. His family are encouraging people to sign a petition to get the "life-changing" Spinraza drug onto the Pharmaceutical Benefits Scheme (PBS).

Wanting to give their son the best possible care, the Dani family relocated from their Unanderra home to Caroline Springs in Victoria just over two years ago.

It breaks my heart when he says why why aren’t my legs working. I just want to walk and play soccer with [brother] Ben.

Shendita Dani

The move was made primarily to be closer to hospitals.

“We were also told that Adem was going to be part of a drug trial because he has type 2 SMA. That unfortunately didn’t happen,” Mrs Dani said.

But her spirits lifted when a drug that can extend the life of children and adults with SMA was approved by the Therapeutic Goods Administration last November.

The new treatment, nusinersen (brand name Spinraza), works by causing the body to increase production of a protein called survival motor neurons, which is critical to the function of muscles.

It is unclear how much the drug will cost, although in the US it can cost $US750,000 ($978,000) for one course of treatment - five to six doses over the first year.

Spinal Muscular Atrophy Australia has launched a petition in Parliament to try and fast track the approval of the new treatment Spinraza on to the Pharmaceutical Benefits Scheme.

Mrs Dani hopes the drug will be added to the PBS because it will be “life-changing for Adem as it will slow the progression” of the devastating degenerative disease.

“Dying from a rare disease is horrible and tragic...it's so hard knowing there is something out there that can help your child but you can't do anything to access it,” she said.

“So many children will die from this horrific disease.

“We just want something to make our son’s life better even if it slows the progression and makes him a bit stronger so he can lift his hands and give me a hug.

“It breaks my heart when he says why why aren’t my legs working. I just want to walk and play soccer with [brother] Ben.”