Treatment for spinal muscular atrophy (SMA) has undergone a major paradigm shift. Today, disease-modifying treatments help patients achieve unprecedented motor milestones, improved motor function, and nutritional and respiratory independence.¹

Early treatment is strongly associated with better outcomes. A 2019 working group of expert physicians and scientists in the United States recommended early treatment for patients with SMA and up to 4 copies of survival motor neuron 2 gene (SMN2).²

Newborn screening is available in all 50 states and Washington, DC, covering 100% of babies born in the United States^3,*

Patients can be identified via newborn screening and diagnosed through genetic testing before symptoms of SMA appear, giving them the best possible chance to benefit from early treatment.^4,5

A 2024 working group of health care providers who are experts in SMA care, recommends immediate confirmation of an SMA diagnosis following a positive newborn screening result. Prompt referral to a center that specializes in SMA treatment was also recommended.¹

*As of January 2, 2024.

Genetic testing is the only way to confirm an SMA diagnosis

The Novartis Laboratory Testing Program can assist with preinfusion lab testing including genetic testing/confirmation and AAV9 antibodies. For more information, please contact your Novartis Regional Account Associate Director or call Novartis Patient Support™ at 1-855-441-4363.

To maximize potential outcomes, SMA experts recommend immediate treatment for all patients with up to 4 copies of SMN2. Delivering treatment before significant motor weakness or neuronal loss occurs may lead to better patient outcomes. Presymptomatic patients may have the most to gain from early treatment.^1,2,5