SMA Australia


Phone: (03) 9796 5744

parliment letter2


PBAC Decision- 15th December 2017

Spinal Muscular Atrophy Australia Inc. is greatly disappointed by the Pharmaceutical Benefits Advisory Committee (PBAC) decision to give Spinraza ‘not a positive’ recommendation for all types of SMA.

Whilst we realise this is devastating news for the entire SMA community here in Australia, rest assured that SMA Australia will continue to play a very active role in this matter on behalf of those who suffer from SMA.

We are encouraged by PBAC’s understanding of the value of this treatment, and their willingness to continue to work with stakeholders towards a more positive outcome as soon as possible. Indeed, we have already attended urgent meetings with the PBAC Deputy Chair and Consumer Representative and the company, Biogen.

SMA Australia will continue to work closely with the PBAC to identify what further information is required. At this early stage, the PBAC has requested more detail about the patient population here in Australia, especially patient numbers and variations. We therefore ask as a matter of urgency that all those who suffer SMA and are not members of SMA Australia to register at the SMA Patient Registry. This will allow us develop a clear picture of SMA in Australia.

The patient registry is located below.

SMA Australia will be highlighting our disappointment in this decision to the Health Minister, and we would greatly appreciate if you too could update any politicians you are in contact with.

Your assistance in these two matters will assist SMA Australia and add weight to continued discussions with the PBAC. We are available for further discussions as to the next steps and can be contacted on 03 9796 5744.

Kind regards,

Julie Cini
SMA Australia Inc.


Want to stay in touch with whats happening with the new treatment for SMA?

This page is for updates and actions for the community to assist to see the drug commercialised here in Australia.

SMA Australia will be advocating at government level to raise awareness about SMA and Spinraza, and we would love the SMA community to get behind this effort.

Register your interest below to be kept up to date. Any enquiries, please do not hesitate to give us a call and have a chat. (03) 9796 5744

SMA Patient Registry


UPDATE 8th June 2017

Thank you to all those who have organised to take a booklet to their local federal politician! There has been some great feedback and positive responses! So thank you. The more we can lift the profile of SMA and raise awareness the better! In light of this we are now hosting an event at Parliament House in Canberra on Wednesday 16th August 2017 ( please see the above invitation. Here we hope to raise the profile and awareness about SMA to our government.

We warmly invite you to attend this event and would live to see as many families as possible in Canberra in August for awareness month. We hope that placing SMA in the spotlight will lead to improved care for all Australians living with SMA, and that the community and government will join us in our mission to progress treatment options, research and timely diagnosis.



November 2016

During the last 12-18 months there has been some great advances in the treatment of Spinal Muscular Atrophy.

Biogen/ Ionis have now released end data for the Type 1 clinical trials, and the investigators have reported that the infants receiving nusinersen experienced significant improvement of motor milestones compared with those who did not receive the treatment. As a result of the findings, Biogen has exercised an option to develop and commercialize nusinersen globally.

This is great news for families all over the world who have been waiting in anticipation for the release of these studies and after releasing this, Biogen realize the urgency of the SMA community and are working to bring this treatment to families.

The SMA Type 1 studies have now stopped and patients will transition into an open label study (this means they know what they are being given) of nusinersen.

Other studies for Type 2 patients will continue in order to collect required data, to demonstrate the safety and efficacy of nusinersen in these populations.


What does this mean for the SMA community here in Australia?

This is great news for families living with SMA here in Australia, as the treatment drug is heading towards being commercialised. We must note that this is the next step in a process for the treatment of SMA. Biogen is just applying to commercialize the drug, it is NOT a guarantee. SMA Australia will be assisting in the next step to work out patient numbers within Australia.

Initially we are asking our families to register with us so you are able to be informed and educated about recruitment for the treatment.

We are working with other stakeholders in updating the SMA patient registry so that better data can be collected and gain a better shop shot of what SMA looks like here in Australia.