New Treatments & Clinical Trials for Spinal Muscular Atrophy

Spinal muscular atrophy is a genetic disease that weakens muscles throughout the body. Three FDA-approved treatments — nusinersen, onasemnogene abeparvovec, and risdiplam — now change the disease course by increasing SMN protein, the protein missing in SMA. Early treatment, especially before symptoms appear, leads to the best outcomes.

What's actually going on in research

Trials are testing combination therapies that pair existing SMN-increasing drugs with treatments for muscle strength, metabolism, and motor function. Researchers are studying gene therapy approaches that may work longer or require less frequent dosing than current options. Studies also focus on adults with SMA, who have fewer treatment options proven in their age group, and on treatments that address muscle wasting and respiratory function directly.

What to know before you search

Eligibility typically depends on SMA type, age, current symptoms, prior treatments, and whether genetic testing confirms SMN1 deletion or mutation.

What types of trials are currently open

• SMN therapy trials — Testing new drugs or formulations that increase SMN protein, the core deficiency in SMA.
• Combination trials — Pairing an approved SMN drug with a muscle-building or metabolic therapy to see if outcomes improve beyond what one drug achieves.
• Gene therapy trials — Testing one-time gene therapies that deliver a working SMN1 gene, aiming for durable benefit.
• Muscle and motor trials — Testing drugs that directly target muscle strength, motor neuron health, or respiratory function.
• Natural history studies — Following people with SMA over time to understand disease progression and establish benchmarks for measuring treatment effects.