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What treatments are available for SMA? 

For many years, there were no medicines available for SMA. However, innovation over the past decade has transformed the SMA landscape as families now have access to treatments.

Three medicines are currently available in Australia that can help slow or stop the progression of SMA. These treatments may also improve muscle function and life expectancy, though their long-term effects are still being studied.

While there is no cure for SMA, treatment focuses on managing symptoms and improving quality of life. A multidisciplinary healthcare team will support you throughout your journey with SMA.

More information on treatments can be found at our Treatments page.

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How is it decided which SMA treatment is the best for each patient? 

Your neurologist will develop a treatment plan that suits you or your child’s individual needs. 

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Will the treatment need to be given for life? 

Since there is no cure for SMA, treatment to manage the condition will be ongoing, unless advised otherwise by your healthcare team. Some available medicines will only require one dose. Further information on treatment can be found at our Treatments page.

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How much does treatment cost? 

The Pharmaceutical Benefits Scheme (PBS) subsidises the three medicines that are currently available in Australia. Patients must be eligible for the PBS and meet the relevant restriction criteria.  

The cost of other treatment options, such as physical therapy to help manage the condition, varies greatly.  

Your healthcare team can provide you with advice on the cost of treatment, as well as information on options for assistance. 

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Beyond medication, what other treatments should people living with SMA consider? 

Physical therapies are an important part of SMA treatment. They can support muscle health and help people navigate the everyday challenges they face when living with SMA. It is especially important for children with SMA, as starting treatment early and following the treatment plan closely can help them reach their full potential. 

Working with a multidisciplinary healthcare team is also important for managing SMA and may include specialists, such as a dietitian. 

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Will there be new treatments for SMA in the future? 

New treatments for SMA continue to be researched, developed, and evaluated through clinical trials. SMA Australia advocates for the latest overseas-approved treatment options to be made available to Australians living with SMA and will keep the community updated as access expands.

Clinical trials – research studies that assess the safety and effectiveness of new medical treatments, tests or procedures in people – may be available for you or your child.  Your neurologist can advise on relevant trials, or you can explore current worldwide clinical trials at ClinicalTrials.gov and discuss potential options with your neurologist.

For a broader view of SMA drug development, the Cure SMA (US) website provides a detailed pipeline of medicines at various stages, from pre-clinical discovery to FDA approval and availability in the United States. You can explore the SMA Drug Pipeline here.

In Australia, the Therapeutic Goods Administration (TGA) oversees medicine approvals, ensuring treatments meet rigorous standards for safety, efficacy, and quality before becoming available to the public. While some medicines gain TGA approval soon after receiving FDA approval, others may face a longer process due to factors such as local clinical trials and additional data requirements – or may not receive TGA approval at all.

Once approved by the TGA, a treatment can be submitted to the Pharmaceutical Benefits Advisory Committee (PBAC) for evaluation. Medicines that receive a positive PBAC recommendation and are subsequently listed on the Pharmaceutical Benefits Scheme (PBS) become accessible for eligible patients in Australia.