5 New Treatments for Spinal Muscular Atrophy That Are Changing Lives

Spinal muscular atrophy (SMA) is a rare but serious genetic disorder that affects the motor neurons responsible for muscle movement. Historically, it was considered a devastating diagnosis with limited treatment options.

But in recent years, medical advancements have led to a new era of hope. Researchers and biotech companies have developed innovative therapies that are dramatically improving outcomes and quality of life for people with SMA.

1. Zolgensma – One-Time Gene Therapy

Zolgensma (onasemnogene abeparvovec-xioi) is a breakthrough gene therapy approved by the FDA for children under 2 years old with SMA. It works by replacing the faulty or missing SMN1 gene responsible for producing the motor neuron protein.

Administered as a single IV infusion
One of the most effective treatments for infantile-onset SMA
Shown to significantly improve survival, motor function, and quality of life

Though expensive, many insurance providers and patient assistance programs help cover the cost.

2. Spinraza – Ongoing Intrathecal Therapy

Spinraza (nusinersen) was the first FDA-approved treatment for all types of SMA. Administered via spinal injection, it boosts production of the SMN protein by modifying how the SMN2 gene is spliced.

Suitable for both children and adults with SMA Types 1–3
Improves muscle strength and motor milestones
Requires loading doses followed by maintenance every four months

It’s widely used and remains a cornerstone of long-term SMA management.

3. CRISPR-Based Genetic Editing – The Most Surprising Frontier

In a groundbreaking twist, researchers are now exploring CRISPR-Cas9 to permanently correct the genetic mutation that causes SMA. Still in preclinical and early-stage trials, this experimental therapy could offer a one-time, lifelong correction at the DNA level.

Targets and repairs the SMN1 gene directly in affected cells
Early results in mice and cell models show dramatic improvements in muscle strength
Could eliminate the need for lifelong treatment

While not yet FDA-approved, this gene-editing technology holds astonishing promise and could become the most revolutionary SMA therapy in years to come.

4. Evrysdi – The At-Home SMA Pill

Evrysdi (risdiplam) is the first oral medication for SMA and is FDA-approved for patients 2 months and older. It works similarly to Spinraza but in a convenient liquid form that can be taken daily at home.

Increases SMN protein production via the SMN2 gene
Suitable for all SMA types, including adult patients
Especially beneficial for those who want to avoid spinal injections

It’s helping democratize treatment access for families and adults who previously struggled with clinic-based therapies.

5. Stem Cell Transplantation – A Regenerative Approach

Although still experimental, stem cell therapy is being studied as a regenerative treatment for SMA. The idea is to replace or regenerate damaged motor neurons with healthy ones derived from stem cells.

Research is ongoing in animal and early human trials
Hopes to provide functional restoration of muscle control
May be used in combination with gene therapies in the future

If proven effective, stem cells could form part of a multi-pronged approach to reversing SMA damage.

Conclusion

SMA treatment has advanced more in the last decade than in the entire history of the condition. From game-changing gene therapies like Zolgensma and oral treatments like Evrysdi to the unexpected promise of CRISPR and stem cell regeneration, these new breakthroughs are turning a once-fatal diagnosis into a manageable condition—and even a reversible one in the future. If you or someone you love is affected by SMA, staying informed about these innovations could make all the difference.

Sources

FDA – https://www.fda.gov

Zolgensma – https://www.zolgensma.com

Spinraza – https://www.spinraza.com

Evrysdi – https://www.evrysdi.com

NIH Clinical Trials – https://www.clinicaltrials.gov

Muscular Dystrophy Association – https://www.mda.org