By Cam Lucadou-Wells
Berwick mother Julie Cini has made good her pledge to her dying daughters.
Ms Cini has celebrated the listing of a life-saving “miracle” medicine for Spinal Muscular Atrophy on the Pharmaceutical Benefits Scheme from 1 June.
Ahead of the 2018-’19 budget, the Federal Government announced $241 million to make the drug Spinraza affordable to 160 families.
Ms Cini’s two babies Montanna and Zarlee succumbed to type-one Spinal Muscular Atrophy many years before the drug was available.
Since then, for the past 14 years, she has lobbied for treatments and support.
“I know my two children … obviously don’t have any benefit from this drug,” Ms Cini said at the emotional 6 May announcement at Royal Childrens’ Hospital Melbourne.
“But I promised them that when they died that I would fix it for everyone else.”
The drug Spinraza which is the only known treatment for the cruel muscle-wasting disease cost patients a prohibitive $367,000 a year.
With the PBS listing, each script will cost patients a maximum of $39.50. Concession patients pay just $6.40.
The PBS listing will be available for SMA patients under 18.
Ms Cini who founded the charity Spinal Muscular Atrophy Australia will now lobby to make the PBS listing available to adults.
Health Minister Greg Hunt said the listing would be “life-saving” and “life-changing” for hundreds of young patients and families.
“It delivers hope for so many beautiful young patents and their families.”
“It’s not about the money. It’s about the hope, the potential and the possibility.”
He thanked Spinraza’s manufacturer Biogen for opening up compassionate access to the drug from 1 May up until the PBS listing.
SMA is an inherited genetic disease that destroys muscles and motor neurons, particularly in the torso.
It slowly deprives children of the means to swallow and breathe. In the case of type-1 SMA patients, few of them reach the age of 2.
The medicine can slow the disease and in some cases, halt its progression.
“If we can get the drug early enough, we can prevent it altogether.”
That means with pre-natal testing funded by the Federal Government, there is hope that the disease can be prevented in all new cases.
Ms Cini has founded a Hallam-based support charity Spinal Muscular Atrophy Australia.
She has personally counselled countless SMA families through their heart-breaking ordeals, but now she can sell hope.
Recently, she travelled to Canberra to successfully lobby the Pharmaceutical Benefits Advisory Committee to recommend PBS listing for types 1, 2 and 3a.
Initially the committee was only set to recommend listing for type 1.
“There’s been blood, sweat and more than enough tears.
“There’s now so much excitement in the SMA space.”
