Muscular atrophy drug is access to hope

Kaitlyn Offer

For little Chloe O'Neill and her family, affordable access to a life-changing spinal muscular atrophy drug is access to hope.

Spinraza will be made available on the PBS from June for patients under 18, Federal Health Minister Greg Hunt announced on Sunday.

Chloe, 10, was diagnosed with SMA type 2 when she was one, was in and out of hospitals between the ages two and four, and has been whizzing around in her electric wheelchair ever since.

And while she has not held her back - "Chloe goes to a normal school, has a huge bunch of friends all supportive of her, they get up to trouble they get up to fun", says her dad Scott - SMA will see her degenerate further.

"Chloe has no ability to shift her weight at all, so sitting in a chair or a couch she's got no ability to sit herself up or sit herself down so she has to ask us to help shift her into position," Mr O'Neill told AAP.

"Even during sleep she'll call out and we'll have to turn her over in her bed."

Chloe will be eligible for Spinraza, which would have cost more than $367,850 a year for the medicine but it will now be $39.50 per script with concessional patients paying $6.40.

It should slow down degeneration in cases like Chloe's.

"It means a lot for us, it changes the future," Mr O'Neill said.

"Today's announcement brings forward hope and it now makes it credible, it's no longer this 'one day' or 'this dream' it's now a reality."