Spinal Muscular Atrophy
Drug Prices & Savings

SMA is a rare genetic disease affecting motor neurons. Three FDA-approved disease-modifying therapies exist — including Zolgensma at $2.1 million, the most expensive drug ever approved.

Spinal Muscular Atrophy Medications & Prices

Compare retail costs, GoodRx prices, and manufacturer patient assistance programs (PAP) for spinal muscular atrophy drugs.

Prices are approximate retail estimates. Actual costs vary by pharmacy, location, and insurance. Always verify with your pharmacist.

Frequently Asked Questions

Common questions about Spinal Muscular Atrophy medications, costs, and savings programs.

What is spinal muscular atrophy (SMA)?
SMA is a rare genetic disease caused by SMN1 gene mutations, leading to motor neuron loss controlling muscle movement. Untreated SMA Type 1 is the most common genetic cause of infant death. Three disease-modifying therapies now exist.
How much does Zolgensma cost?
Zolgensma is priced at $2.1 million — the most expensive drug in the world. It's a one-time IV gene therapy providing a functioning SMN1 gene. Novartis offers a managed access program; contact your SMA specialist for enrollment.
What is the difference between Spinraza, Evrysdi, and Zolgensma?
Spinraza is an intrathecal injection given 4 times per year. Evrysdi is an oral daily liquid. Zolgensma is a one-time IV infusion at $2.1M. All increase SMN protein — Zolgensma adds a new gene; Spinraza and Evrysdi modify SMN2 splicing.
Does Biogen have a patient assistance program for Spinraza?
Yes. Biogen's SMA360 program provides copay assistance and free drug for eligible uninsured patients. Most SMA patients need specialty pharmacy and insurance navigation support. Contact 1-800-456-2255.
What is newborn screening for SMA?
Newborn screening for SMA is standard in 42+ US states as of 2024. Early identification before symptom onset is critical — infants diagnosed and treated pre-symptomatically often achieve normal motor milestones.

Newly Diagnosed with Spinal Muscular Atrophy?

A quick-start guide to understanding your diagnosis and managing medication costs.

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Understand Your Treatment

Ask your doctor about first-line vs. specialty medications, expected timeline, and monitoring requirements. Write down all questions before appointments.

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Check for Patient Assistance

Before filling any prescription, ask your pharmacist about manufacturer copay cards, PAP programs, and GoodRx pricing. Many specialty drugs are free or near-free for qualifying patients.

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See a Specialist

For complex conditions, a specialist (rheumatologist, neurologist, pulmonologist, etc.) provides access to newer therapies and clinical trials unavailable at the primary care level.

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Connect with Others

Patient advocacy organizations offer peer support, educational resources, and can connect you with financial assistance navigators who specialize in your condition.

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Public Voices on Spinal Muscular Atrophy

Many public figures live with spinal muscular atrophy and speak openly about their experience.

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