By Counterforce Health Team —

Zolgensma (Onasemnogene Abeparvovec): A Breakthrough Gene Therapy for SMA

Zolgensma (generic name: onasemnogene abeparvovec) is a groundbreaking gene therapy used to treat spinal muscular atrophy (SMA) — a rare, inherited condition that causes progressive muscle weakness and loss of movement in infants and young children.

Approved by the U.S. FDA in 2019, Zolgensma is one of the most advanced treatments in modern medicine, offering hope to families affected by this devastating disease.

What Is Spinal Muscular Atrophy (SMA)?

SMA is caused by a missing or faulty SMN1 gene, which is responsible for producing a vital protein called survival motor neuron (SMN). Without enough SMN protein, motor neurons in the spinal cord deteriorate, leading to muscle weakness and difficulty breathing, swallowing, and moving.

How Zolgensma Works

Zolgensma delivers a healthy copy of the SMN1 gene into the body’s cells using a harmless, modified virus (AAV9). This new gene enables the body to make the missing SMN protein, helping protect and restore muscle function.

Unlike other SMA treatments that require ongoing doses, Zolgensma is given as a single one-time infusion — designed to address the root genetic cause of the disease.

How It’s Given

Zolgensma is administered through an intravenous (IV) infusion over about one hour in a hospital or specialized medical center.

After treatment, patients are closely monitored for several months with regular blood tests to check liver function and platelet counts. Because the therapy can temporarily affect the liver, children also receive oral corticosteroids for a short period after infusion.

Common Side Effects

Most side effects are temporary and manageable. The most common include:

  • Elevated liver enzymes
  • Vomiting
  • Low platelet counts

Careful medical monitoring before and after treatment helps prevent and manage potential complications.

Benefits of Zolgensma

Zolgensma has changed the outlook for children with SMA. Clinical trials show that many treated infants:

  • Achieve motor milestones such as sitting, crawling, or walking
  • Gain strength and improved breathing function
  • Experience slower or halted disease progression compared to untreated children

Early diagnosis and treatment — ideally before symptoms appear — can lead to the best outcomes.

A Hopeful Future

Zolgensma represents a major step forward in gene therapy and rare disease treatment. For families facing an SMA diagnosis, it offers something once thought impossible — a chance for a healthier, more active childhood.

If your child has been diagnosed with SMA, talk with your neurologist or genetic specialist about whether Zolgensma may be an option.